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If you know the name of a drug manufacturer, you can easily search using the alphabetical menu below. Or you can search in free text – simply type the disease, drug, company name, or other detail into the Search bar below and click on the magnifying glass. For broadest results, type the terms without quotation marks; to narrow your search to an exact match, put your terms in quotation marks (i.e.; “breast cancer”). See Search Tips for tips and definitions to make your searches more effective.
The web links in this directory are public information provided to the Foundation and the acknowledgement times listed are the number of days or hours for a company to acknowledge receipt of request, not to make a decision. Data contained in this directory is provided either directly by the company or by ClinicalTrials.gov for the companies listed in this directory.
The Expanded Access listings provided on this website are limited to those companies currently in the Company Directory and include listings for expanded access which are Available, Temporarily Not Available and Approved for Marketing, which in some cases includes treatments which have not yet reached the market. In addition, many companies are willing to consider expanded access requests even if they do not show active Expanded Access Listings in this directory. Contact the company to explore the possibility of individual access to an investigational therapy.
To learn more about clinical trials and expanded access, please visit our patient and physician guides on this website.
4SC
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
4SC focuses on the development of novel small molecule drugs for the treatment of cancer in indications with a high unmet medical need. Such drugs are intended to provide patients with innovative treatment options that are more tolerable and efficacious than existing therapies and provide a better quality of life. 4SC’s current product pipeline includes two programs in clinical development: resminostat and domatinostat.
We understand the interest of patients in accessing resminostat or domatinostat outside of clinical trials and prior to regulatory approval. However, participation in one of our clinical trials is currently the most appropriate way to access our investigational therapies. 4SC has decided not to offer Expanded Access (EA) to any of our investigational products at this time and will not approve EA requests.
We recognize the need for Expanded Access programs, and we will re-evaluate the status of our policy based on data from ongoing and future clinical trials.
If you have additional questions, please consult your physician or contact medical.request@4sc.com. We anticipate acknowledging receipt of requests sent to this email within five business days.
Abbisko Therapeutics Co., Ltd.
Single-Patient EA Policies/Criteria
Abbisko aspires to discover and develop differentiated therapeutics for the treatment of cancer diseases. One of the Abbisko’s lead products, ABSK021, is an oral, potent, highly selective CSF-1R inhibitor. It is currently being investigated as a single agent in one Phase 3 clinical trial for the treatment of patients with TGCT (Tenosynovial Giant Cell Tumor). We are conducting clinical trials aiming at obtaining regulatory approval to make ABSK021 available to all patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access ABSK021 at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at clinicaltrials.gov..
Abbisko understands the interest of patients in accessing ABSK021 outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases, however we do not currently offer any Expanded Access Programs for ABSK021.
Abbisko recognizes the need for Expanded Access Programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.
Abbvie
Expected Application Timeframe
2 business daysABM Therapeutics
Expected Application Timeframe
10 Business DaysSingle-Patient EA Policies/Criteria
This is the physician-sponsored, single-patient extend access (EA) to ABM-1310. ABM-1310 is an investigational small molecule inhibitor for treating patients with BRAFv600 mutant malignancies. This program currently is only available through licensed physicians in the United States and is offered to those patients who previously participated in an ABM-1310 clinical trial in the U.S., to allow them to continue receiving ABM-1310 therapy. ABM Therapeutic (ABM) is the ABM-1310 drug provider and may provide possible assistance to the physicians for their EA applications.
Physician applicants are responsible for submitting required documents and obtaining all required approvals prior to requesting ABM-1310 drug supply. ABM reserves the right to review, approve or reject EA applications based on case-by-case evaluation. The ABM-1310 drug supply is free of charge to treating physicians and patients after all approvals are granted. All patient care is the responsibility of the treating physician and the patient’s own healthcare coverages.
Available Therapies via Single-Patient EA
The ABM-1310 single-patient EA is specifically for patients in the U.S. who previously participated in an ABM-1310 trial and continued ABM-1310 treatment when the trial ended.
This EA is only for patients with advanced BRAFv600 mutant solid tumors including primary brain tumors.
AC Immune SA
Available Therapies via Single-Patient EA
AC Immune SA, a clinical-stage biopharmaceutical company, is a global leader in developing precision medicine for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and certain rare indications.
AC Immune has determined that given the stage of development of its products, its investigational drugs should be studied in patients as part of controlled clinical trials designed to obtain data on safety and efficacy that may be used to support approval of the product and subsequent wider accessibility to patients. We encourage patients to speak with their physicians and to participate in the available clinical trials. At this time, AC Immune cannot provide investigational drug outside of clinical studies. The company will reevaluate this policy when sufficient safety and efficacy information has been obtained in controlled clinical trials.
Accendatech USA Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Our EA policy will apply to those DMG patients who exhausted standard of care and can't find appropriate trials to participate. We encourage a patient's provider (pediatric neuro-oncologist) to reach out to us so that we can discuss the case with the provider before an EA decision can be made based on the clinical data we collected so far and the information from each patient.
Available Therapies via Single-Patient EA
ACT001 for DMG treatment
Achillion Pharmaceuticals
Expected Application Timeframe
N/AAvailable Therapies via Single-Patient EA
The mission of Achillion is to provide better treatments for people with serious diseases. We are a science-driven company committed to putting patients first. In our endeavor to bring innovative medicines to patients as fast and safely as possible, we conduct clinical trials to establish the safety and efficacy of an ‘investigational drug’. An ‘investigational drug’ is a potential medicine that is in active clinical development, but has not yet received marketing approval by regulatory authorities. Clinical trials and the subsequent timely generation of safety and efficacy data are the most effective way of ensuring review and decision making by Regulatory Authorities. This ultimately has resulted in access to new, safe and effective approved medicines for patients.
Achillion supports the need for Early Access to investigational drugs for patients ineligible to enroll in clinical trials.
An Early Access program for a specific investigational drug may be opened if Achillion determines that all the following criteria are met:
1. The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
2. An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
3. Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
4. There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient
Achillion continues to assess the eligibility requirements and criteria for Early Access to the investigational drug danicopan (ACH-4471). At this time, an Early Access Program (EAP) is not available. We will re-evaluate this policy from time to time.
ADC Therapeutics
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
If you cannot be treated by currently available drugs, cell therapy, or clinical trials, contact your treating physician to determine if the loncastuximab tesirine EAP is an option.
Available Therapies via Single-Patient EA
A U.S. EAP for loncastuximab tesirine (ADCT-402) in Relapsed or Refractory Diffuse Large B Cell Lymphoma (R/R DLBCL).
AffyImmune Therapeutics, Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
AffyImmune is committed to developing next-generation T cell therapies for cancer patients who have serious and life-threatening diseases or conditions.
Our Expanded Access Policy (EAP) refers to the use of an investigational cell therapy outside of a clinical trial. Sometimes called “compassionate use”, expanded access is a potential pathway for a patient to gain access to investigational therapies in certain rare circumstances in which a person has no other available therapies or is not eligible to participate in a current AffyImmune clinical trial. At AffyImmune, we recognize and understand the need for an expanded access policy for patients who have serious or immediately life-threatening disease and have limited available treatment options.
The decision to establish an EAP is dependent on several key factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ guidelines. These include, but are not limited, to the following:
• The patient or patients to be treated have a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
• The potential patient benefit justifies the potential risks of using the treatment and those potential risks are not unreasonable in the context of the disease or condition to be treated; and
• Providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use.
AffyImmune believes that participation in one of our clinical trials is the best way to access our investigational cell-based gene therapies. We encourage patients to speak with their physicians regarding participating in clinical trials. In rare cases where patients with serious diseases are unable to participate in clinical trials and have exhausted all available options, AffyImmune may consider providing our investigational therapies outside of a clinical trial. As a general policy, AffyImmune will not provide our investigational therapies until sufficient preliminary safety and efficacy information has been obtained in our clinical trials, typically following Phase 2 investigation.
If you are a patient who is interested in accessing our investigational cell therapies, please speak with your physician. You may also learn more about ongoing clinical trial(s) by going to www.clinicaltrials.gov and searching for AffyImmune.
If you are a physician who is interested in learning more about our investigational cell therapies, or participating in our clinical trials, please submit a request to clinicaltrials@AffyImmune.com. We will use our best efforts to respond within 3 business days after receipt of your request.
Available Therapies via Single-Patient EA
The treatment that is available via this single-patient expanded access is AIC100, a CAR T product currently in a clinical trial in patients with relapsed or refractory thyroid cancer.
AiCuris Anti-infective Cures AG
Expected Application Timeframe
N/ADisease/Category-Specific EA Policies/Criteria
AiCuris develops Pritelivir oral tablets for the treatment of acyclovir-resistant mucocutaneous herpes simplex virus (HSV) infections. A phase 2 trial in immunocompromised patients is ongoing in the US. AiCuris supports expanded access requests for Pritelivir oral tablets for the treatment of eligible patients and collaborates with myTomorrows to facilitate early access to Pritelivir for acyclovir-resistant mucocutaneous HSV infections in immunocompromised patients.
To get access, patients need to consult with their treating physician to explore all treatment options. If the physician agrees to a treatment with Pritelivir oral tablets and to oversee the patient's treatment, the physician should contact the myTomorrows medical team for more information on this EAP. When providing medical information about the patient, please note that only anonymized data shall be included in any patient information. Due to EU data protection law this shall not be accompanied by personal data.
Akero Therapeutics, Inc
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
Although Akero retains sole discretion in deciding whether to grant an expanded access request, the following criteria will guide evaluation of requests:
• The condition or disease being studied is serious or life-threatening and there are no available alternative treatments;
• The patient is not eligible for an ongoing or planned Akero-sponsored clinical trial;
• There must be sufficient clinical data to identify an appropriate dose for the proposed use;
• The potential benefit to the patient clearly outweighs the collective potential risks;
• There must be adequate supply of the investigational product to meet the needs of the expanded access program without impacting the company’s clinical trials;
• The patient meets any other relevant medical criteria for expanded access to the investigational product, as determined by Akero.
https://akerotx.com/clinical-trials/
Available Therapies via Single-Patient EA
Efruxifermin (EFX) for the treatment of nonalcoholic steatohepatitis (NASH)
Akeso Biopharma, Inc
Single-Patient EA Policies/Criteria
Akeso Biopharma, Inc. (Akeso) is a biopharmaceutical company committed to bringing new, innovative therapies to patients by conducting rigorous clinical trials and obtaining marketing approval by the U.S. Food and Drug Administration (FDA), China National Medical Products Administration (NMPA) and other regulatory authorities. Akeso conducts clinical trials to study investigational medicines in patients in which the studies are designed to obtain data on safety and efficacy that may be used to support approval of the product and subsequent wider accessibility to patients. Investigational medicines are drugs or biologics that have not been approved or cleared by regulatory authorities.
Akeso seeks to retain the ability to manufacture and supply investigational medicines in a fair and equitable manner and in a volume that assures adequate supply for ongoing clinical trials and development programs. Hence, at this time, Akeso is unable to provide its investigational medicines on an expanded access or right to try basis. For patients seeking access to our investigational medicines before they are approved by a regulatory authority, participation in one of Akeso’s clinical trials is the most appropriate way to access these investigational medicines. To learn more about available clinical trials by Akeso, please visit https://www.akesobio.com, or visit https://clinicaltrials.gov and search by company, disease or medicine.
If you have additional questions, please speak with your physician or contact Akeso at clinicaltrials@akesobio.com.
Consistent with the 21st Century Cures Act, Akeso may revise this policy at any time.
Alexion
Expected Application Timeframe
3 business daysAlkermes
Expected Application Timeframe
2 weeksSingle-Patient EA Policies/Criteria
http://www.alkermes.com/assets/content/files/Expanded%20Access%20Policy.pdf
AlloVir, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
AlloVir is committed to developing safe and effective therapies addressing severe and potentially life-threatening virus-associated diseases and their potential sequelae. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Expanded access refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.
A number of factors consistent with the US Food and Drug Administration (US FDA) and other regulatory agencies’ guidelines should be taken into account when considering expanded access. They include:
• The illness must be serious or immediately life-threatening with no other satisfactory treatment options (such as approved products or enrolling clinical trials).
• The patient must have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• The patient is ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• There is an ability to provide the product in a fair and equitable manner and there is adequate manufacturing capacity for ongoing programs.
• Whether granting expanded access would potentially compromise the scientific validity of broader development programs or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients.
At this time, AlloVir believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. If you have additional questions, please speak with your physician or contact expandedaccess@allovir.com. We anticipate acknowledging receipt of requests sent to this email within five business days.
Alnylam Pharmaceuticals
Expected Application Timeframe
3 business daysDisease/Category-Specific EA Policies/Criteria
http://www.alnylam.com/medical-professionals/expanded-access-program/
Alpine Immune Sciences, Inc.
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
Alpine will evaluate an expanded access request based on a variety of factors, which include but are not limited to:
• The request for expanded access is submitted by a treating physician, who is appropriately licensed;
• The patient’s condition is serious or life-threatening and there is a reasonable potential that the investigational drug has the potential to provide a benefit to the patient with an acceptable level of risk;
• Participation in a clinical trial is not an option, either because the patient is not eligible to enroll or because there are no appropriate ongoing clinical trials;
• The proposed dose of the investigational drug is within the existing dose range for which human safety data are available;
• The investigational product is available for treatment use without compromising supplies that have been designated for other uses.
Requests will be considered on a case-by-case basis. If a request for expanded access is granted, the requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring, and safety reporting.
Available Therapies via Single-Patient EA
ALPN-101
ALX Oncology Inc
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
At ALX Oncology, our focus is on the discovery and development of novel anti-cancer compounds that block the CD47 - SIRPα pathway. The goal of our current clinical study program is to enroll patients and obtain clinical data on ALX148 that is required by the US Food and Drug Administration (FDA) and other regulatory authorities for review and approval of marketing applications for this investigational agent. We believe that focusing on clinical studies is the most appropriate way to achieve this goal; therefore, we do not currently offer access to ALX148 outside of clinical studies and, at this time, we have no Expanded Access program (sometimes referred to as “compassionate use” or “pre-approval access”).
For information on participating in and status of ALX148 clinical trials, please see: https://clinicaltrials.gov/ct2/home
If you have additional questions regarding this policy, please speak with your health care provider or contact: info@alxoncology.com
Amgen
Expected Application Timeframe
3 business daysAmicus Therapeutics
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Requests for Early Access cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver. Patients and caregivers seeking general information may reach out to Amicus’ Global Patient & Professional Advocacy Department at patientadvocacy@amicusrx.com or call toll-free in the United States at 1-866-9AMICUS (1-866-926-4287), or +44-1753-888-567 for International inquiries.
Amryt Pharmaceuticals
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
The Company has not implemented an expanded access program (EAP) in the US for use of AP101 in epidermolysis bullosa (EB). There is an ongoing Phase 3 randomised controlled study evaluating the safety and efficacy of AP101 for the treatment of patients with junctional and dystrophic EB. While the study is ongoing and equipoise remains with respect to the benefits and risks for AP101 in this patient population, the Company’s policy is to encourage all potentially eligible patients in the US to be considered for enrolment in the clinical trial
Angiocrine Bioscience, Inc.
Single-Patient EA Policies/Criteria
At this time, Angiocrine’s investigational therapies are not available on an expanded access or right-to-try basis for new patients. In the event Angiocrine decides to consider expanded access or right-to-try use, the Company will update its policy at that time and then evaluate and respond to each request that it receives on a case-by-case basis. For more information on our investigational therapies and ongoing clinical trials, please visit clinicaltrials.gov.
Applied Therapeutics Inc.
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
At Applied Therapeutics, we recognize that some patients with serious or immediately life-threatening diseases may not be able or eligible to participate in clinical studies and may not have other treatment options for their condition. Applied Therapeutics evaluates requests for individual patients to receive investigational therapies outside of a clinical study on a case-by-case basis. Requests for expanded access must be made by the patient’s physician.
Available Therapies via Single-Patient EA
AT-007 (gavorestat)
Disease/Category-Specific EA Policies/Criteria
https://www.appliedtherapeutics.com/at-007-expanded-access-program/
Aravive, Inc
Expected Application Timeframe
5-10 business daysSingle-Patient EA Policies/Criteria
Aravive, Inc. is a clinical-stage biopharmaceutical company developing treatments designed to halt the progression of life-threatening diseases, including cancer. The following is Aravive’s policy for evaluating and responding to requests for individual patient access to its investigational drugs that are intended to treat serious diseases.
Aravive believes that investigational drugs should be studied in patients as part of clinical trials designed to obtain data on safety and efficacy that may be used to support approval of the product and subsequent wider accessibility to patients. We encourage patients to speak with their physicians and to participate in clinical trials. In rare cases when patients with serious diseases are unable to participate in clinical trials and have exhausted all available therapies, Aravive may consider providing an investigational drug outside of a clinical trial.
General Criteria
We consider a number of factors when determining whether or not to support expanded access for a given investigational drug:
Will the potential benefit potentially outweigh the collective potential risks to the patient?
Is there adequate drug supply for both the clinical development program (current and future trials) and broad expanded access?
Is there enough evidence to reasonably assess that the drug will be safe and effective so that it can be provided to patients under expanded access in an environment that is less controlled than a sponsored clinical trial?
To meet regulatory requirements, will the safety data be adequately provided to Aravive by a local physician outside of a sponsored clinical trial?
Is there a good understanding of the indication for which use is requested?
Will it jeopardize the ongoing development work that Aravive is conducting to bring a therapeutic to market as quickly as possible and to as many patients as possible?
In addition, the program must be compliant with local rules and laws and the treating physician has to be willing to open a single-patient (Investigator) IND with the FDA.
Contact Information
A treating physician may submit questions or requests regarding expanded access to the following: clinicaltrials@aravive.com
Additional information may be obtained from the U.S. Food and Drug Administration at https://www.fda.gov/downloads/newsevents/publichealthfocus/expandedaccesscompassionateuse/ucm504494…
Request Procedures
Requests should be submitted to clinicaltrials@aravive.com by the treating licensed physician and should include sufficient supporting detail to enable Aravive to evaluate the expanded access request. Please include contact information so Aravive can follow-up with the physician directly (i.e., address, phone number, e-mail). The requesting physician would be expected to submit an Investigator IND, if appropriate, only after Aravive review of the provided information and approval of the request.
Aravive may revise this expanded access policy at any time. Additionally, the posting of the policy by Aravive shall not serve as a guarantee of access to any specific investigational drug for any patient.
Available Therapies via Single-Patient EA
AVB-500 is a therapeutic recombinant fusion protein that has been shown to neutralize GAS6 activity by binding to GAS6 with very high affinity. In doing so, AVB-500 selectively inhibits the GAS6-AXL signaling pathway. Aravive reported positive data from the first 31 patients enrolled in the Phase 1b portion of a Phase 1b/2 clinical trial of AVB-500 in platinum-resistant recurrent ovarian cancer. AVB-500 continues to be well tolerated. Investigator-sponsored Phase 1/2 trials of AVB-500, in combination with durvalumab in patients with platinum-resistant recurrent epithelial ovarian cancer and with avelumab in patients with advanced urothelial Carcinoma (COAXIN), are also ongoing.
Disease/Category-Specific EA Policies/Criteria
Arcellx, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
At this time, Arcellx's investigational therapies can only be accessed through participation in a clinical trial. Additional details regarding Arcellx's active clinical trials can be found at www.clinicaltrials.gov.
Available Therapies via Single-Patient EA
Currently, we have no therapies available via single-patient EA.
Disease/Category-Specific EA Policies/Criteria
argenx
Expected Application Timeframe
5 daysArmaGen
Expected Application Timeframe
N/AArrowhead Pharmaceuticals, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Arrowhead requires the following criteria be met for a request to be considered:
• The investigational medicine must be part of an active clinical development program,
• Access to the medicine will not compromise clinical trials or the regulatory pathway,
• There is substantial scientific evidence to support the benefit risk profile of the investigational product for its intended use,
• Sufficient supply of the investigational medicine is available, and
• It is logistically possible to safely administer the investigational medicine outside of a clinical trial setting
The patient and the treating physician must also meet the following criteria to be considered by Arrowhead for access to an investigational medicine:
• He/she has a serious or immediately life-threatening disease
• He/she lacks other currently available therapeutic options
• He/she is unable to join an active clinical trial of the investigational product
• His/her treating physician believes there is potential for the patient under consideration to reasonably expect benefit from the treatment
Available Therapies via Single-Patient EA
ARO-AAT Injection
For the treatment of alpha-1 antitrypsin deficiency associated liver disease.
Disease/Category-Specific EA Policies/Criteria
https://arrowheadpharma.com/wp-content/uploads/2019/03/Compassionate-Use-Expanded-Access.pdf
Artugen Therapeutics
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
At this time, we do not have an expanded access program that allows patients to have access to our investigational products prior to FDA approval.
ASC Therapeutics, Inc.
Single-Patient EA Policies/Criteria
Currently, ASC Therapeutics does not offer an expanded access program. We are early in the development and are still evaluating the risks associated with its investigational gene therapy, mainly related to immune-related side effects at administration, assessment of the optimal dose, understanding of the duration of treatment response, and long-term loss of some or all treatment effects. For these reasons, ASC Therapeutics has decided not to provide expanded access to its investigational gene therapy until there is clarity on these issues, a decision made after thorough consideration and in consultation with our hemophilia advisory board. Currently, access to our investigational therapy is possible only through participation in clinical trials.Disease/Category-Specific EA Policies/Criteria
https://www.asctherapeutics.com/expanded-access-policy/Ascendis Pharma A/S
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
Overview
- The decision to seek access for treatment utilizing the EAP must be initiated by the patient’s physician. Ascendis Pharma will not incentivize the healthcare provider (HCP) in any way for accessing any Investigational Treatment. The HCP will have sole responsibility and accountability for the clinical management and treatment of the patient.
- Compliance Considerations:
- Requests for EAP use must comply with local standards of care, along with the laws and regulations that govern the use of any Investigational Treatment.
- An HCP request to access EAP on behalf of a patient may entail disclosure of personal patient medical information for assessment by Ascendis Pharma in order to assess eligibility. All patient information must be redacted so that no personally identifiable information is transmitted.
- Requests require both HCP to initiate the request as well as signed consent by the patient.
Criteria for Evaluating Individual Access
HCP Criteria and Responsibilities:
Must be currently licensed in his/her respective jurisdiction and must not ever have been convicted of a criminal offence relating to any therapy, food, or cosmetic law.
HCP must not be on the current FDA debarment list or that of any of the relevant local health authorities in the relevant jurisdiction.
Provide all necessary paperwork to Sponsor for routing to an Investigational Review Board (IRB) or Ethics Committee (EC) as appropriate in their relevant health authority’s jurisdiction.
The HCP must agree to oversee the patient’s treatment and supervise administration of the Investigational Therapy in accordance with Ascendis Pharma’s defined access criteria and under appropriate regulatory and ethical standards including those set by regulatory authorities and IRBs/ECs.
The HCP must agree to obtain informed consent and agreement for treatment use of the Investigational Therapy, and collection, analysis, publication, and transfer of any patient data as appropriate from the patient or his/her legal guardians.
The HCP must agree to maintain and release to regulatory authorities all treatment records and data as required by local regulations.
The HCP must agree to report adverse events and provide patient level safety data as required by local health authorities and per the agreement with Ascendis Pharma
Patient Eligibility Criteria:
- A patient may obtain access to an Investigational Therapy for treatment use via EAP only through a licensed HCP.
- Generally, patient eligibility for consideration of treatment with Investigational Therapy include, but not limited to:
- Patient must be diagnosed with either a serious, debilitating, or life-threatening medical condition.
- A clearly documented biological or clinical rationale should support the potential clinical benefit to the patient.
- Suitable treatment options are not available to treat the ongoing condition OR the patient has a documented attempt at current standard of care but has not responded to treatment.
- The patient does not have an option to participate in a relevant Ascendis sponsored clinical trial because of non-eligibility or the trial is not conducted in the patient's region.
- The patient must have continued access to the HCP for ongoing care.
Request Process
Requests for EAP must be made by the patient’s treating physician.
Available Therapies via Single-Patient EA
Palopegteriparatide (also known as TransCon PTH) for patients with hypoparathyroidism
Ascentage Pharma
Expected Application Timeframe
5-7 business daysAvailable Therapies via Single-Patient EA
This program is not available in the United States of America.
Olverembatinib is a novel third generation BCR-ABL inhibitor that can effectively target a spectrum of BCR-ABL mutants, including the T315I mutation. Olverembatinib is approved in China for the treatment of adult patients with tyrosine kinase inhibitor (TKI)-resistant chronic phase chronic myeloid leukemia (CP-CML) or accelerated-phase CML (AP-CML) harboring the T315I mutation as confirmed by a validated diagnostic test.
Astellas Pharma US
Expected Application Timeframe
7 daysAstraZeneca
Expected Application Timeframe
Immediately via auto-reply emailSingle-Patient EA Policies/Criteria
Where there is no MPEAP, AstraZeneca may make an investigational medicine available to a single patient in accordance with country-specific regulations. Terminology and requirements of named patient access schemes vary globally; in the US, for instance this includes the FDA’s guidelines for treatment of individual patients with an Investigational New Drug (IND). (Please review criteria listed on the EA Policy document.)
Early Access requests submitted via the online Early Access request platform and e-mails submitted to EarlyAccess@AstraZeneca.com will be acknowledged immediately upon receipt via an automated email reply.
AstraZeneca Pharmaceuticals LP
Phone Number & Email
Expected Application Timeframe
ImmediateAtossa Genetics
Expected Application Timeframe
1-5 daysSingle-Patient EA Policies/Criteria
Atossa Genetics is a clinical-stage biopharmaceutical company developing novel, proprietary therapeutics and delivery methods for breast cancer and other breast conditions.
Atossa Genetics’ development resources are focused on conducting clinical studies to fully answer important scientific questions about the potential risks and benefits of the investigational products, and to obtain marketing approval by the FDA and other regulatory health authorities.
Atossa Genetics is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Atossa Genetics’ investigational products by contacting the Company.
The purpose of this policy is to describe the requirements for Expanded Access to Atossa Genetics investigational products to patients outside of a clinical study.
ATOSSA GENETICS’ APPROACH TO REQUESTS FOR ACCESS TO EXPERIMENTAL THERAPIES
• Participation in clinical trials is the first and most preferable route.
• If participation in clinical trials is not an option, physicians may consider other options, which may include expanded access programs managed by the company or single-patient expanded access.
• Requests are considered on a case-by-case basis in a fair and equitable manner.
GENERAL CRITERIA USED TO EVALUATE ACCESS REQUESTS FOR INDIVIDUAL PATIENTS
The patient has a serious or life-threatening condition with no satisfactory alternative.
• Assessment that benefits outweigh the risks to the patient.
• Assessment that the company has an adequate supply of the investigational medicine.
• A determination that expanded access will not interfere with the company’s ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients.
Available Therapies via Single-Patient EA
Oral endoxifen (for pre-surgical and adjuvant treatment of Luminal A ER+ breast cancer; mammographic breast density)
Disease/Category-Specific EA Policies/Criteria
https://www.atossagenetics.com/wp-content/uploads/2019/07/AG-Expanded-Access-Policy.pdf
Atox Bio
Expected Application Timeframe
2 business daysSingle-Patient EA Policies/Criteria
Atox Bio is not accepting requests for expanded access at this time. The ongoing clinical trial is the most appropriate and expeditious way to advance therapies to patients. The ongoing trial can be viewed at clinicaltrials.gov.
Disease/Category-Specific EA Policies/Criteria
Atsena Therapeutics, Inc.
Single-Patient EA Policies/Criteria
Participation in Atsena Therapeutics’ clinical trials is currently the only pathway for patients to gain access to our investigational therapies. We are not currently offering expanded access to any of our investigational therapies.
Attralus, Inc.
Expected Application Timeframe
7 Business DaysSingle-Patient EA Policies/Criteria
At this time, participation in one of our Attralus clinical trials or investigator-sponsored clinical trials is the only way to access any Attralus investigational products. For more information regarding clinical trials with Attralus investigational therapies or diagnostics, please visit www.clinicaltrials.gov.
Expanded access requests will be referred to study investigators to determine whether a patient is eligible for any ongoing clinical trials. If a patient does not qualify for one of our ongoing clinical trials, referrals may also be provided to patient organizations and/or academic centers who may be able to identify other appropriate clinical trials or approved treatment options.
Available Therapies via Single-Patient EA
None at this time.
Avalo Therapeutics, Inc.
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
Avalo will consider requests from licensed treating physicians for access to investigational products in development for patient treatment purposes on a case-by-case basis.
Treating physicians interested in potential access to investigational products may request more information by contacting Avalo via email at expandedaccess@avalotx.com. Please note that only requests submitted by treating physicians will be considered.
Receipt of a request for information will be acknowledged within 10 business days.
Some of the factors that will be considered upon receiving a request include:
- Whether the patient has a serious or life-threatening illness
- Whether the patient has undergone standard treatment without success or there is no viable alternative therapy
- Whether the potential benefits of the experimental product will outweigh any potential risk to the patient
- Whether the patient is ineligible for or is unable to participate in a clinical trial for the investigational product
- The impact of providing access to an investigational product on the development program for that product
- The impact of providing access to an investigational product on the available inventory and supply for that product
Avalo’s decision on whether or not to grant expanded access to the investigational product under an Expanded Access Program should be communicated to the requestor within 15 business days of receipt.
Application under this policy is not a guarantee of access to any Avalo investigational drug. Avalo reserves the right to revise or revoke this policy at any time.
Additionally, Avalo reserves the right to deny any request at it’s discretion.
Decisions for expanded access will be made on a fair and equitable basis for each investigational therapy. Each disease we are studying may have different specific criteria due to the differences in urgency and known data on safety and efficacy.
Available Therapies via Single-Patient EA
AVTX-801
AVTX-802
AVTX-803
AVTX-002
Disease/Category-Specific EA Policies/Criteria
https://www.avalotx.com/patients-and-families/access-to-investigational-therapies
Avelas Biosciences, Inc.
Single-Patient EA Policies/Criteria
At Avelas Biosciences, we are committed to developing products that bring new, innovative therapies, like pegloprastide (AVB-620), to patients with serious or life-threatening illnesses or conditions. Avelas is developing pegloprastide to improve the ability of surgeons to identify cancerous tissue on the surface of tissue during breast cancer surgery. Pegloprastide must be used in conjunction with a specific imaging device to visualize potentially cancerous tissue during surgery.
At this time, Avelas does not offer an expanded access program and is not accepting expanded access requests for investigational products, such as pegloprastide.
Avelas’ current focus and priority is to complete the product development program for its investigational product(s), such as pegloprastide, in order to obtain the required safety and efficacy data needed for regulatory approval. We believe that focusing our resources on our clinical trial program is the best path forward to bring our investigational product(s) to patients as quickly and safely as possible.
As we continue to develop investigational product(s), we will review our expanded access policy for investigational product(s) and may make updates to this policy.
Patients can gain access to our investigational product(s), such as pegloprastide, by participating in a clinical trial. If you or someone you know would like to learn more about Avelas Biosciences’ clinical trials, we encourage you to view our trials at www.clinicaltrials.gov.
If you have additional questions about Avelas’ expanded access policy, please email us at info@avelasbio.com.
Avenge Bio
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
At our current stage of development, Avenge Bio does not have an active expanded access program that allows patients to have access to our investigational products prior to FDA approval. Avenge Bio may revise this expanded access policy at any time as we advance the development of our product and will make available the specific requirements to access the program.
AVM Biotechnology
Expected Application Timeframe
48 hoursSingle-Patient EA Policies/Criteria
The purpose of our compassionate use program is to make AVM0703 available to those patients who have exhausted other treatment options and who do not meet the criteria to enroll in a clinical trial. It is intended to improve access to AVM0703 for patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options and may benefit from it.
Available Therapies via Single-Patient EA
AVM0703 Injection is a novel and proprietary formulation of high concentration (24 mg/mL) of dexamethasone.
It is the subject of two clinical investigations:
• Leukemia and Lymphoma (ClinicalTrials.gov Identifier: NCT04329728)
• ARDS (Acute Respiratory Distress Syndrome) mediated by COVD-19 or Influenza (A or B) (ClinicalTrials.gov Identifier: NCT04366115)
Disease/Category-Specific EA Policies/Criteria
https://avmbiotech.com/wp-content/uploads/2020/09/AVMExternal-EAP.pdf
Avobis Bio, LLC
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
At this time, Avobis Bio is offering an Expanded Access Program on a limited basis pursuant to Avobis’ Expanded Access Protocol. To ensure the safety of patients, we have developed criteria to evaluate applicable Expanded Access Program requests. Any such requests must be in accordance with laws and regulations and our internal policies and procedures. Further, we cannot guarantee access to any investigational drug by any individual patient. FDA guidelines note that Expanded Access may be appropriate when the below criteria are met:
1. Patient has a serious disease or condition, or whose life is immediately threatened.
2. There is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition.
3. Enrollment in a clinical trial is not possible.
4. Probable patient benefit justifies the probable risks of treatment with investigational drugs.
5. There is sufficient evidence of safety and effectiveness to support the use of the investigational drug.
6. Providing the investigational medical product will not affect the investigational trials.
7. The Expanded Access request is made by a physician who is qualified and licensed in accordance with State law and who is knowledgeable in the treatment of the disease or condition.
A treating physician must initiate an Expanded Access request. If you are a patient or caregiver, please reach out to your doctor to assist you in this process. Physicians should send Expanded Access requests to expandedaccess@avobisbio.com. The physician requesting Expanded Access must provide specific information supporting the request, including:
1. A copy of the requesting physician’s curriculum vitae (CV).
2. A copy of the physician’s current medical license, which must be in good standing in the state where the therapy will be administered. By submission of a medical license, the requesting physician attests they are not under any investigation or threatened loss of privileges to practice.
3. Relevant patient history, excluding any individually identifiable health information or protected health information (PHI).
4. Scientific rationale supporting the potential benefits of the investigational product based on available clinical and non-clinical data.
5. Statement that there is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the patient’s disease or condition.
6. Statement that the patient has exhausted all approved therapy options, or that the patient is no longer responsive to or able to tolerate such therapies.
We will acknowledge receipt of a physician’s request within five business days.
Medical and scientific professionals at Avobis Bio who are familiar with the available data for the investigational product will evaluate all applicable requests on an individual basis in accordance with the criteria above, pursuant to Avobis Bio’s Expanded Access IND and using the scientific evidence available at the time of the request and other information relevant to patient safety to aid their evaluation.
Posting this policy by Avobis Bio does not guarantee access to any specific investigational drug for any individual patient. We encourage patients to speak with their physicians about the possibility of enrolling in clinical trials in order to gain access to investigational therapies. Additionally, patients can search www.clinicaltrials.gov for information regarding clinical trials. If you have questions about our Expanded Access Program, please contact us at expandedaccess@avobisbio.com
Available Therapies via Single-Patient EA
AVB-114 is an autologous investigational product under evaluation in Seeded Cells on Matrix Plug Treating Crohn's Perianal Fistulas (STOMP-II; a prospective, multicenter, randomized, blinded outcome assessment, controlled, add-on therapy phase II study) for the treatment of complex perianal fistulas in subjects with Crohn's disease. EAP-STOMP2 is an expanded access protocol for patients who had adipose tissue collected per the STOMP-II protocol, but who were subsequently unable to receive AVB-114 treatment due to screen failure or product manufacturing issues and thus withdrew from the study. Patients who withdrew from STOMP-II for other reasons will not be considered for EAP-STOMP2. This EAP will only be considered at Clinical Investigative Sites who enrolled subjects in STOMP-II.
Inclusion Criteria
In order to be eligible to participate in this EAP, an individual must meet all of the following criteria:
1. Provision of signed and dated ICF.
2. Adipose tissue previously collected per the STOMP-II protocol, but were
subsequently unable to receive AVB-114 treatment due to screen failure or product manufacturing issues and thus withdrew.
3. Diagnosed with Crohn’s disease and a target perianal fistula with one internal
opening and at least one external opening.
Exclusion Criteria
An individual who meets one or more of the following criteria will be excluded from participation in this EAP:
1. Suffer from any condition or illness that, in the opinion of the Investigator, would compromise patient safety.
2. Pregnant, trying to become pregnant, or are breast feeding. Women of childbearing potential (WCBP) and males who have sexual partners that are WCBP must agree to use an adequate method of contraception while participating in this EAP.
3. Known allergies or hypersensitivity to aminoglycosides.
4. History of clinically significant fat-directed autoimmunity.
5. Active, unresolved infection requiring parenteral antibiotics.
6. Unresolved abscess at time of treatment (if screening Pelvic MRI identifies an
abscess, it must be resolved before treatment).
7. Target fistula with any of the following characteristics (note that presence of a second fistula tract that is distinctly separate from the target fistula, or a target fistula with a single internal opening and multiple external openings is not exclusionary):
a. Target fistula has a branching blind ending sinus tract (no external opening) that investigator assesses to be at elevated risk of abscess formation if treated with AVB-114.
b. Target fistula is a genito-urinary fistula, including rectovaginal (i.e., fistulae that
transverse the vaginal canal).
c. Any anatomical limitation to successfully securing the fistula plug disk.
Bayer
Expected Application Timeframe
Two working daysSingle-Patient EA Policies/Criteria
If your doctor believes that an investigational medicine could help you but you cannot take part in a clinical trial, it might still be possible to access the treatment. Single patient access may not be allowed in some countries, so please check your local regulations to find out. This is only possible in exceptional circumstances when you, or someone you’re caring for, are suffering from a severely debilitating or life-threatening disease that cannot be treated with available approved drugs, or where all possible approved treatments have been unsuccessful. At this point, your doctor might consider an investigational treatment as the only way to help.
We will let your doctor know we have received the application within two working days. Once we have all the information we need, we will do our best to complete our assessment and get back to your doctor within five working days.
We can only agree to provide an investigational medicine to your doctor if we agree with their medical evaluation that the possible benefits of the drug outweigh the potential risks. We take care to ensure that we comply with the laws that apply in different countries for investigational medicines and treatments. We also work together with doctors to ensure that they comply with local laws regarding access to investigational medicines, including monitoring and recording patient safety information. We consider every request for access carefully, but we cannot guarantee that it will be granted.
Beacon Therapeutics (Formerly AGTC)
Single-Patient EA Policies/Criteria
At Beacon Therapeutics, we are committed to developing products that bring new, innovative gene therapies, like AGTC-501 (laruparetigene zovaparvovec), to patients with serious diseases with no or limited treatment options.
Currently, Beacon Therapeutics does not offer an expanded access program and is not accepting expanded access requests for our investigational products, including AGTC-501.
We understand patient interest in accessing AGTC-501 outside of clinical trials and prior to regulatory approval. However, participation in one of our clinical trials is currently the most appropriate way to access our investigational products. Please see ClinicalTrials.gov and the Beacon Clinical Trials webpage for publicly available information related to Beacon Therapeutics’ ongoing clinical trials.
If you have any questions about Beacon Therapeutics’ EA policy, please contact expandedaccess@beacontx.com
Belite Bio, Inc.
Expected Application Timeframe
7 daysSingle-Patient EA Policies/Criteria
Belite Bio is currently unable to support an Expanded Access to its investigational drug (Tinlarebant, aka, LBS-008) due to very limited supplies of drug product.
Available Therapies via Single-Patient EA
None of Belite Bio's investigational drug products are available through Expanded Access.
Disease/Category-Specific EA Policies/Criteria
BerGenBio ASA
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
BerGenBio ASA is a clinical-stage oncology company focused on developing transformative drugs targeting AXL as a potential cornerstone of therapy for aggressive diseases, including immune-evasive, therapy resistant cancers. BerGenBio is currently developing a novel investigational medicinal drug product, bemcentinib (BGB324). Bemcentinib is in the clinical stage of development, being evaluated as a treatment option in COVID-19 and various oncology settings, including acute myeloid leukaemia (AML) and non-small cell lung cancer (NSCLC).
At BerGenBio, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval, making bemcentinib available to patients as quickly as possible. As such, we believe participation in our clinical trial is the most appropriate way to access bemcentinib at this stage of development.
BerGenBio understands the interest of patients in accessing bemcentinib outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases. However, we do not offer any of our developmental medicines through expanded access programmes, with the exception of patients who could benefit for continued treatment upon completion of a company sponsored trial. We encourage those who are interested in learning more about our clinical development programmes, including eligibility criteria and locations to visit clinicaltrials.gov.
Available Therapies via Single-Patient EA
At BerGenBio, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval and making bemcentinib available to patients as quickly as possible. As such, we believe participation in a company-approved clinical trial is the most appropriate way to access bemcentinib at this stage of development. BerGenBio understands the interest of patients in accessing bemcentinib outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases. However, we do not offer any of our developmental medicines through expanded access programs, except for patients who could benefit for continued treatment upon completion of a company-approved trial.
Binhui Biopharmaceutical Co., Ltd
Expected Application Timeframe
10 Business DaysSingle-Patient EA Policies/Criteria
Binhui Bio currently has only one clinical drug product that can be used for EA, which is OH2 for the treatment of patients with unresectable stage III or IV melanoma that are resistant to anti-PD1 therapy.
Available Therapies via Single-Patient EA
Binhui Bio, a leading biotechnology company specializing in the research, development, and pilot production of innovative drugs, places patient wellbeing at the forefront of our priorities. Among our breakthroughs, the OH2 injection, a unique product derived from oncolytic viruses, has garnered attention by securing five INDs and one orphan drug approval from China NMPA and US FDA since 2018. The OH2 injection, designed for monotherapy and combination therapy for advanced melanoma cancer.
Recognizing that there may be scenarios where patients require access to our yet-to-be-approved medicines beyond the scope of clinical trials, we have established guidelines to address these unique situations. Such exceptions might arise when patients battling serious or life-threatening diseases have no other means to join a clinical trial, and lack satisfactory alternative treatment options. Under these circumstances, a healthcare professional (HCP) may be granted the use of an unlicensed medicine for patient treatment. This kind of utilization of unapproved medicines outside the framework of clinical trials is commonly referred to as "compassionate use" or "expanded access".
This policy delineates Binhui's approach to the treatment use of our unlicensed pharmaceutical products and the criteria we employ to assess requests from HCPs.
• The patient's condition being addressed is either life-threatening or causes significant impairment to daily life.
• There are no other satisfactory treatment options available, as confirmed by the treating HCP.
• Sufficient evidence is available that indicates the potential benefits of the unlicensed medicine for the patient outweigh the potential risks involved.
Moreover, we consider several additional factors when making decisions about supplying unlicensed medicines:
• There should be enough detailed information available to guide the correct application of the investigational medicine, particularly concerning the appropriate dosage and duration of treatment for the patient.
• We assess the potential impact of the treatment use of the investigational medicine on any concurrent clinical trials or its regulatory pathway.
• We only allow the use of our medicines in countries with an adequate medical infrastructure to safely administer the treatment.
• We only extend use in broad access programs to countries where Binhui Bio intends to pursue regulatory approval and subsequently make the medicine available. This condition may not necessarily apply to individual-named patients.
• We consider the ethical implications of our decisions, with particular attention to the principle of fair treatment for all patients.
• We ensure that the proposed use of our treatment is in accordance with local legal and regulatory frameworks.
Our senior medical staff, strictly following medical and ethical criteria, makes decisions to supply unlicensed medicines for treatment use.
We at Binhui Bio strongly believe that participation in one of our clinical trials is the most effective way to access our OH2.
HCPs seeking to request unlicensed medicines for treatment use should file the treatment IND to FDA and submit relevant information to IRB.
For further information on submitting a request for treatment use of our investigational medicines, HCPs are directed to follow this link. https://www.binhui-bio.com/
Bioatla, Inc
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
Expanded Access is a potential pathway for patients diagnosed with a serious and/or life-threatening disease or condition to gain access to an investigational product for treatment outside of a clinical trial when no comparable or satisfactory alternative therapy options are available. In these instances, a patient’s treating physician can request an investigational product prior to regulatory approval, where allowed by local laws.
Who is Eligible for Expanded Access Programs?
BioAtla considers granting expanded access to its investigational products when all of the following criteria are met:
• A patient is diagnosed with a serious and/or life-threatening disease or condition.
• There are no adequate standard or investigational therapies available.
• The patient is not eligible for or cannot participate in a BioAtla-sponsored study for the therapy being requested.
• There must be adequate data supporting use of the investigational medication in the treatment of the patient’s
disease.
• Adequate supply exists to support both the ongoing clinical trials and expanded access, until and if a product
becomes commercially available.
• Expanded access will not adversely impact the clinical development program or the regulatory approval process.
• The request must be made, unsolicited, by the patient’s treating physician.
• The treating physician must also agree to comply with the safety and monitoring requirements.
• Where appropriate demonstration of efficacy and safety may be required for continued treatment.
• Expanded access is permitted or approved in the country, region, and/or state where the investigational medicine
will be administered.
• Any condition, in addition to those listed above, that in the opinion of BioAtla may preclude appropriate use of the
investigational medicine, will be considered.
Available Therapies via Single-Patient EA
A Phase 2 Open Label Study of BA3021 in Patients With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
Biogen
Expected Application Timeframe
1 business dayBiohaven Pharmaceuticals
Expected Application Timeframe
2 business daysSingle-Patient EA Policies/Criteria
Requests for Early Access must be made by a physician on behalf of their patient. A licensed physician overseeing the patient’s care, who is able to comply with Biohaven’s requirements, may contact Biohaven at BHV0223.ExpandedAccess@earlyaccesscare.com or call toll-free in the United States at 1-888-315-5797 (Option 6).
Bionomics Limited
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
Bionomics Limited does not offer an expanded access program for its investigational products.
BioNova Pharmaceuticals (Shanghai) Limited
Expected Application Timeframe
14 Business DaysAvailable Therapies via Single-Patient EA
BioNova Pharmaceuticals (Shanghai) Limited (hereinafter referred to as BioNova) is currently not providing BN104 Tablets for Expanded Access use.
BN104 is a novel, highly selective and potent oral menin inhibitor developed by BioNova and currently being investigated as a potential treatment for acute leukemias patients who harbor a mixed-lineage leukemia rearranged (MLLr) or nucleophosmin (NPM1) mutation. On October 23, 2023, BN104 was designated as a Fast Track product by the FDA.
A phase 1 clinical study constitutes the Part 1 dose-escalation and Part 2 dose-optimization will be conducted in eligible patients with acute leukemias. The study is designed to establish a safe and optimized dose and appropriate dosing schedule for BN104. Once RP2D established, and with agreement from the FDA, the company will initiate the phase 2 program in patients with acute leukemias who harbor a MLLr or NPM1 mutation.
BioNova will review its EA policy annually to determine if a change in policy is warranted and feasible.
Disease/Category-Specific EA Policies/Criteria
BioNova will respond to all Expanded Access requests within 14 business days. All requests for Expanded Access or additional information may be sent by email to info@bionovapharma.com. For email requests, please indicate EXPANDED ACCESS REQUEST in the subject line.
Biosight
Single-Patient EA Policies/Criteria
Biosight is a clinical stage biotech company, developing innovative therapeutics for hematological malignancies and disorders. Biosight’s lead product, BST-236 (INN aspacytarabine), is an innovative proprietary anti-metabolite designed to enable high-dose therapy with reduced systemic toxicity. BST-236 is currently being investigated as a single agent in a phase 2b clinical trial as a first-line treatment of acute myeloid leukemia (AML) in patients unfit for standard chemotherapy. Additional studies in patients with relapsed or refractory AML and myelodysplastic syndrome (MDS) are under preparation.
At Biosight, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval, making BST-236 available to patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access BST-236 at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at www.clinicaltrials.gov.
Biosight understands the interest of patients in accessing BST-236 outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases, however we do not currently offer any Expanded Access programs for BST-236.
Biosight recognizes the need for Expanded Access programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.
Biosolution Co.,Ltd
Phone Number & Email
Expected Application Timeframe
10 Business DaysSingle-Patient EA Policies/Criteria
CartiLife is an autologous chondrocyte cell-based therapy for the restoration of symptomatic and structural complications caused by articular cartilage defects.
Suitable for treatment of defects which are: ICRS grade III or IV, with defect sizes between 2 ~ 10 cm^2.
Available Therapies via Single-Patient EA
Biosolution Co.,Ltd does not provide access to investigational products outside of clinical trials. We encourage patients to participate in clinical trials of our investigational therapies whenever possible, because clinical trials are designed, conducted, and monitored to ensure that the safety and efficacy are appropriately evaluated before they are submitted to regulatory agencies for review with the intent to make them more broadly available to patients.
Biosolution Co.,Ltd
Phone Number & Email
Expected Application Timeframe
10 Business DaysSingle-Patient EA Policies/Criteria
CartiLife is an autologous chondrocyte cell-based therapy for the restoration of symptomatic and structural complications caused by articular cartilage defects.
Suitable for treatment of defects which are: ICRS grade III or IV, with defect sizes between 2 ~ 10 cm^2.
Available Therapies via Single-Patient EA
Biosolution Co.,Ltd does not provide access to investigational products outside of clinical trials. We encourage patients to participate in clinical trials of our investigational therapies whenever possible, because clinical trials are designed, conducted, and monitored to ensure that the safety and efficacy are appropriately evaluated before they are submitted to regulatory agencies for review with the intent to make them more broadly available to patients.
Biothera Pharmaceuticals, Inc.
Expected Application Timeframe
7 business daysSingle-Patient EA Policies/Criteria
Biothera is evaluating the safety and efficacy of its proprietary innate immune modulator, Imprime PGG, in combination with various immune checkpoint inhibitors and monoclonal antibodies for the treatment of multiple cancers. At this time, Biothera does not have a compassionate use program; however, it will consider providing Imprime PGG for individual patients (Single Patient IND) on a case-by-case basis. This opportunity applies only to patients with serious or life-threatening conditions who are unable to participate in a clinical trial and for whom there are no comparable or satisfactory alternative therapy options.
In determining whether single patient access is appropriate outside of a clinical trial, Biothera will consider many factors, including: the strength of available efficacy and safety data; the patient’s medical condition and history; the benefit-risk profile of our drug in relation to the patient’s case; the potential impact on the clinical development program; and the ability and willingness of the patient’s physician to sponsor the treatment application.
If you are a potential patient and your physician believes you may be a candidate for single patient access to Imprime PGG, please ask your physician to contact Biothera at earlyaccess@biothera.com.
Available Therapies via Single-Patient EA
Imprime PGG
BioXcel Therapeutics
Expected Application Timeframe
5 Business DaysBlaze Bioscience, Inc.
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
This is Blaze Bioscience's expanded access policy for its investigational drug tozuleristide intended for patients with life-threatening diseases or conditions who have exhausted approved treatment options and are unable to participate in a clinical trial involving the investigational drug tozuleristide.
1. Contact Information: A licensed treating physician may submit questions or requests on behalf of a patient regarding expanded access to tozuleristide to be evaluated in accordance with Blaze Bioscience company policies. Physician Expanded Access Requests should be submitted in writing to expandedaccess@blazebioscience.com and include “Expanded Access Request” in the subject.
2. Request Procedures:
a. General Criteria: Blaze Bioscience will evaluate and respond to each Expanded Access Request individually and on a case-by-case basis. Criteria Blaze Bioscience will use in its evaluation of whether to grant Expanded Access Request include:
1) Adequate supply of the investigational drug tozuleristide must be available above and beyond the supply needed for Blaze Bioscience clinical trials;
2) There is sufficient clinical data to identify an appropriate dose of the investigational drug;
3) There is a good understanding of the patient’s clinical situation and investigational drug proposed use for surgery including the proposed fluorescence detection device;
4) All available therapeutic approaches for the patient’s disease have been exhausted by the patient and their physicians;
5) The investigational drug is considered an “eligible investigational drug” under Section 561(B)(2) of the FDCA at the time of the Expanded Access Request;
6) Providing the investigational drug is compliant with all applicable rules and laws;
7) Appropriate Institutional Review Board/Ethics Committee and FDA authorization requested expanded access has been obtained;
8) Treating physician understands and is willing to be responsible for ensuring that the patient informed consent requirements are met; and
9) Treating physician understands and is willing to be the holder of a treatment IND with FDA.
b. Timing of acknowledgement: Blaze Bioscience endeavors to acknowledge requests within ten (10) business days of receipt of an Expanded Access Request.
c. Clinical trials: Blaze Bioscience lists its active clinical trials on clinicaltrials.gov. Before granting an Expanded Access Request for tozuleristide, written confirmation by the treating physician that the patient is not eligible for an active Company clinical trial is needed prior to consideration of the Expanded Access Request.
Available Therapies via Single-Patient EA
tozuleristide
Blueprint Medicines Corporation
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Please see our Pre-Approval Access Policy on our website
Available Therapies via Single-Patient EA
Investigational medicine, avapritinib (formerly known as BLU-285)
See also: https://clinicaltrials.gov/ct2/show/NCT03862885?term=avapritinib&draw=2&rank=4
Investigational medicine, pralsetinib (formerly known as BLU-667)
See also: https://clinicaltrials.gov/ct2/show/NCT04204928?term=pralsetinib&draw=2&rank=1
Disease/Category-Specific EA Policies/Criteria
Avapritinib: Advanced gastrointestinal stromal tumors (GIST), advanced or smoldering systemic mastocytosis (SM), or another tumor type with a mutation in exon 17 of the KIT gene or in exon 18 of the PDGFRA gene
Pralsetinib: Advanced Non-Small Cell Lung Cancer (NSCLC) or Medullary Thyroid Cancer (MTC)
BlueRock Therapeutics, LLC
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
BlueRock has initiated a Ph1 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of embryonic stem cell-derived dopaminergic neurons in patients with advanced Parkinson’s disease. We understand that some patients may wish to access our investigational drugs that have not yet been approved. At this stage of drug development and trials, we do not have enough clinical product or evidence of clinical safety and efficacy of our investigational drugs to support expanded access. Therefore, BlueRock Therapeutics does not currently offer an expanded access program at this time. The most appropriate way for patients to access our investigational drug is by participating in our clinical trials, and we encourage you to speak with your doctor about whether you would qualify for this trial. Information regarding ongoing clinical trials can be accessed at www.clinicaltrials.gov. (ClinicalTrials.gov Identifier: NCT0480273)
If you have additional questions about BlueRock’s expanded access policy, please speak to your physician, or contact regulatory@bluerocktx.com.
Boehringer Ingelheim Pharmaceuticals
Expected Application Timeframe
5 business daysBristol-Myers Squibb
Expected Application Timeframe
48 hoursC4 Therapeutics, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
On our website, both physicians and patients may explore information about our investigational agents. Our EAP policy is described below.
Several factors consistent with the FDA and other regulatory agencies’ guidelines, are essential when considering expanded access requests:
- The illness must be serious or life-threatening with no other satisfactory treatment options (such as approved products or open clinical trials)
- There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on available safety and efficacy information
- The ability to provide a therapy in a fair and equitable manner, so that there is adequate manufacturing capacity to provide therapies across all ongoing clinical trials and expanded access programs
- Whether granting expanded access would potentially compromise the scientific validity of broader development programs, or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients
Given the early stage of our development program, we believe that participation in our clinical trial is the most appropriate way to access our investigational therapy. Therefore, we are not currently making our investigational product available through an expanded access program. If you have additional questions, please speak with your physician or have your physician contact clinicaltrials@c4therapeutics.com. We anticipate acknowledging receipt of requests sent to this email within five business days.
In line with the 21st Century Cures Act, C4 Therapeutics, Inc may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on clinicaltrials.gov if such record becomes active.
Cara Therapeutics
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Cara Therapeutics is committed to the development and commercialization of novel therapies for the treatment of chronic pruritic conditions. To do this, we conduct clinical trials to assess the safety and efficacy of our investigational products. For a list of clinical trials currently recruiting patients, please visit www.clinicaltrials.gov.
Clinical trials generate data that may allow us to apply for approval of our therapies for commercialization from regulatory agencies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Regulatory approval of our products may ultimately provide broader access to our medicines.
Celgene
Expected Application Timeframe
24 hoursCelldex Therapeutics
Expected Application Timeframe
5 business daysAvailable Therapies via Single-Patient EA
None
Disease/Category-Specific EA Policies/Criteria
https://celldex.com/docs/Compassionate_Use_Policy_MAY2018.pdf
Cellectar Biosciences
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Cellectar Biosciences is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer. Our core objective is to leverage our proprietary phospholipid drug conjugates™ (PDCs™) delivery platform to develop PDCs that specifically target cancer cells to deliver improved efficacy and better safety as a result of fewer off-target effects.
Consistent with Cellectar Biosciences’ mission to bring our PDCs™ to patients with cancer, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our products available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing. At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Cellectar Biosciences will consider providing a requesting physician with pre-approval access to a specific Cellectar Biosciences investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:
• The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.
Cellectar Biosciences is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Cellectar Biosciences may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Cellectar Biosciences whose decisions are final. Currently available therapies include CLR 131 for 4th line or later relapsed or refractory multiple myeloma and patients with relapsed or refractory diffuse large B-cell lymphomas (DLBCL) who have received at least two prior treatment regimens; and patients with relapsed or refractory lymphoplasmacytic lymphoma (LPL) or Waldenstrom’s Macroglobulinemia (WM) who have received at least two prior treatment regimens.
Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to clinical@cellectar.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 5 business days after receipt.
Available Therapies via Single-Patient EA
CLR 131 4th line or later relapsed or refractory multiple myeloma and patients with relapsed or refractory diffuse large B-cell lymphomas (DLBCL) who have received at least two prior treatment regimens; and patients with relapsed or refractory lymphoplasmacytic lymphoma (LPL) or Waldenstrom’s Macroglobulinemia (WM) who have received at least two prior treatment regimens.
Cellphire Therapeutics, Inc.
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
Cellphire Policy: Expanded Access Use of Thrombosomes®
Overview
This Policy regarding Expanded Access to Cellphire’s Investigational New Drug Thrombosomes (TBX), an activated freeze-dried platelet, is aimed at addressing the limited availability of platelets that are the result of the COVID-19 pandemic. Blood products are in critical need during this time frame and blood banks are faced with extremely limited supplies.
In the situation where a patient presents with WHO Grade 3 bleeding or higher and is in critical need of platelet/blood products, this Policy facilitates the use of TBX to treat the acute bleed and possibly mitigate the risk of additional critical bleeding. The benefit of the use of TBX in this critical set of circumstances is believed to outweigh the risks of the use of this investigational product.
Policy Statements
In cases where a clinical trial with Thrombosomes is not an option, Cellphire may elect to provide the Sponsor/Investigator expanded access to its investigational product, TBX. Treating Sponsor/Investigators and patients should note that clinical safety and efficacy of investigational products has not been fully established, so all potential risks and benefits should be carefully evaluated before seeking expanded access to this product. It is envisioned that requests could be made by Sponsor/Investigators as either an Individual Patient IND or Emergency Use Individual Patient IND. For more information on submitting requests to the FDA, click here.
This policy is aimed at addressing both types of requests. Cellphire will consider requests for access to TBX, according to internal Cellphire SOPs and as permitted by applicable law, in very specific circumstances, when certain criteria are met. For more detailed information, see Cellphire’s full policy here.
Requesting Access
Sponsor/Investigators seeking single patient expanded access (either emergency or non-emergency use) to TBX on behalf of their patient should call Cellphire at 301-545-2528 or submit an inquiry to expandedaccess@cellphire.com.
The request will promptly be addressed, generally within 24 hours of receipt by a member of either the Cellphire Clinical Affairs or Clinical Study Teams. Sponsor/Investigators should provide a written summary of the specific medical circumstances that require single patient expanded access and the need for treatment with TBX which briefly includes responses to each of the eligibility requirements listed above.
It should be noted that there is no guarantee that an expanded access request will be granted by FDA. Sponsor/Investigators who receive a TBX dose for their patients through the expanded access program must comply with all applicable FDA regulations, contractual conditions, safety reporting required by FDA, and protection of intellectual property.
This policy is subject to change. Cellphire will revisit the policy periodically and amend it as appropriate. This policy is not a guarantee of access to any of Cellphire’s investigational products.
Available Therapies via Single-Patient EA
Thrombosomes(R) - where a patient presents with WHO Grade 3 bleeding or higher and is in critical need of platelet/blood products, this Policy facilitates the use of TBX to treat the acute bleed and possibly mitigate the risk of additional critical bleeding.
Center for Cancer Research, National Cancer Institute, National Institutes of Health
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Due to the unique nature of personalized cell manufacturing and the high-level of monitoring required for patients treated with this product, an expanded access program to provide this therapy to patients at their local institution is not feasible at this time. For patients who may fit the indication to receive this therapy, and are between the ages of 3-30 with CD22 positive B-cell Acute Lymphoblastic Leukemia that is refractory or in second or later relapse, and either CD19 negative or relapsed/refractory to CD19 targeting, enrollment may be possible on the ongoing National Institutes of Health CD22 CAR T-cell clinical trial “Anti-CD22 Chimeric Receptor T Cells in Pediatric and Young Adults With Recurrent or Refractory CD22-expressing B Cell Malignancies” (NCT02315612).
Available Therapies via Single-Patient EA
https://clinicalstudies.info.nih.gov/ProtocolDetails.aspx?A_2015-C-0029.html
Checkpoint Therapeutics, Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Checkpoint Therapeutics, Inc. (Checkpoint) is clinical-stage biotechnology company focused on accelerating the development and commercialization of potential life-changing oncology therapeutics. Checkpoint’s lead asset, cosibelimab (CK-301), is under clinical investigation for the potential treatment of solid tumor indications, and a biologics license application (BLA) for cosibelimab is currently under review with the US Food and Drug Administration for the potential approval of cosibelimab for advanced cutaneous squamous cell carcinoma. As clinical investigation of cosibelimab in solid tumors is ongoing, the preferred patient access pathway to cosibelimab treatment is via clinical trial participation. Information about cosibelimab clinical trials, including key eligibility criteria and clinical trial locations, is available at clinicaltrials.gov.
For access to cosibelimab treatment or other Checkpoint investigative therapies outside of ongoing clinical trials and prior to regulatory approval (eg, for patients who do not qualify for or are not able to participate in a current Checkpoint-sponsored clinical trial), Checkpoint will consider a physician’s request for access to a Checkpoint investigative therapy outside of the clinical trial setting. All such requests must be made in accordance with local laws and regulations and must be submitted by the treating physician to checkpointexpandedaccess@checkpointtx.com for Checkpoint’s review.
Available Therapies via Single-Patient EA
Cosibelimab (also referred to as CK-301)
Chiesi USA
Expected Application Timeframe
5 calendar daysSingle-Patient EA Policies/Criteria
Chiesi USA’s stated goals include the development of pharmaceutical solutions to improve the quality of human life and to combine commitment to result with integrity, operating in a socially responsible manner. In line with the Chiesi Mission and our core values, Chiesi USA will consider granting access to patients whenever possible who meet the following criteria:
- The patient has a serious or life-threatening illness with no comparable or satisfactory alternative therapies.
- The patient is ineligible for, or otherwise unable to, participate in a clinical trial related to the Investigational Product requested. Geographical limitations related to investigational product access will not necessarily render expanded access subjects ineligible.
- In the treating physician’s judgement, the potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated.
- The Investigational Product is currently being studied in humans.
- Adequate supply exists or can be produced to support both the ongoing clinical investigations and compassionate use.
- Providing the Investigational Product for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support FDA approval of the expanded access use or otherwise compromise the potential development of the expanded access use.
Available Therapies via Single-Patient EA
Pegunigalsidase alfa for Fabry disease
Chigenovo Co., Ltd.
Expected Application Timeframe
See link for detailsSingle-Patient EA Policies/Criteria
See link for details
Clarity Pharmaceuticals Ltd
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
Currently, Clarity does not have an expanded access program in place. This decision may change once more information on the safety and efficacy of our products become available through clinical trials. As we advance our research and gather more data, Clarity is committed to exploring ways to improve patient access to our investigational products.
Available Therapies via Single-Patient EA
Not applicable. Clarity currently does not have an expanded access program in place.
Codagenix Inc.
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
Codagenix Inc. (www.codagenix.com) is developing investigational vaccines for the prevention of a variety of infectious diseases. Our vaccines are in early stages of development; therefore, their potential risks and benefits are in the process of being evaluated. Participation in clinical trials should be the primary route by which patients obtain access to investigational vaccines and contribute to the collection of safety and efficacy data needed to support regulatory approval worldwide. Because our vaccines by their nature are designed for prophylaxis, not treatment use, Codagenix does not intend to offer Expanded Access to these investigational vaccines. To learn more about available clinical trials, please visit www.clinicaltrials.gov and search using the company name.
Disease/Category-Specific EA Policies/Criteria
Cook MyoSite, Incorporated
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Cook MyoSite is committed to conducting rigorous, controlled clinical trials with variable inclusion and exclusion criteria based upon the indicated use under investigation. These trials are designed to demonstrate to regulatory authorities that our investigational product is safe and effective, and as a result, allow our investigational cell therapy product to become a valuable treatment option for a wide range of patients. Participation in our clinical trials is the first and most preferable route to access our investigational product.
Cook MyoSite will consider providing an investigational product to a qualified requesting physician currently licensed within the United States via the United States Food and Drug Administration’s (FDA’s) expanded access pathway outside of an active clinical trial when the following general requirements are met:
• The patient has a serious or life-threatening disease or condition with no satisfactory alternative;
• Positive assessment that the anticipated benefits outweigh the risks to the patient;
• Positive assessment that Cook MyoSite has an adequate supply of resources for producing the investigational product;
• A determination that expanded access will not interfere with Cook MyoSite’s ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients;
• The patient can undergo a muscle biopsy procedure(s), and;
• The patient can undergo Cook MyoSite’s required donor screening and testing.
For additional information, please refer to our website: https://resources.cookmyosite.com/terms-and-conditions-of-sale-and-delivery-0
Available Therapies via Single-Patient EA
Autologous Muscle Derived cells (AMDC).
Single-patient EA studies have included AMDC for the treatment of esophageal aperistalsis and AMDC for the treatment of underactive bladder
Crestone, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
At Crestone, we are committed to developing CRS3123 as a first-in-class treatment for patients with Clostridioides difficile infection. We are conducting clinical studies to demonstrate safety and efficacy to obtain regulatory approval and ultimately make CRS3123 available. Currently, Crestone does not offer an expanded access program and does not accept expanded access requests outside of clinical trials.
Prior to regulatory approval by the U.S. Food and Drug Administration (FDA) and foreign regulatory authorities, patients gain access to investigational treatments, such as CRS3123, by participating in clinical trials. Expanded access is also referred to as “compassionate use”, “Early Access”, and “Emergency Use”. Patients who seek access to investigational medicines outside of an established clinical study and prior to health authority marketing authorization may wish to do so because standard treatments have failed, they cannot tolerate already approved medicines, because they are unable to participate in a clinical study or because there are no comparable or satisfactory therapy options available outside of clinical trials. The primary purpose of expanded access is to use the investigational drug for patient treatment purposes rather than to gather data on safety, tolerability, and effectiveness.
We believe that access to CRS3123 should be limited to clinical trials until such time as its safety, tolerability and effectiveness for a particular indication has been determined and confirmed by regulatory authorities. Patient access to CRS3123 outside of a clinical trial may interfere with the conduct of our ongoing and future clinical trials, and may also disrupt the progress of our development program, which would, in turn, delay access to many patients in need.
Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials is the only safe and appropriate way to access CRS3123.
If you have questions about Crestone’s expanded access policy, please contact Crestone at drugsafety@crestonepharma.com. Please expect a response within five business days.
In line with the 21st Century Cures Act, Crestone may revise this policy at any time.
CStone Pharmaceuticals (Suzhou) Co., Ltd.
Single-Patient EA Policies/Criteria
CStone Pharmaceuticals (Suzhou) Co., Ltd. (CStone) is a biopharmaceutical company committed to bringing innovative therapies to patients by conducting clinical trials and obtaining marketing approval by the US Food and Drug Administration (FDA), China National Medical Products Administration (NMPA) and other regulatory authorities. Sugemalimab, an anti-PD-L1 monoclonal antibody, is currently investigated in a Phase 2 clinical trial for subjects with relapsed or refractory extranodal natural killer/ T cell lymphoma (R/R ENKTL), as well as other Phase 1-3 trials indicated for a variety of hematologic malignancies and solid tumors.
Expanded Access, which is sometimes known as “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational therapy for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. For more information about expanded access in the US, please visit the FDA website at https://www.fda.gov/news-events/public-health-focus/expanded-access.
Consistent with CStone’s mission, our development resources are focused on conducting clinical trials required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of sugemalimab, and to gain regulatory approvals to make sugemalimab available broadly to patients as quickly as possible. To reach this goal successfully, our top priorities must be to ensure that an adequate supply of sugemalimab is available for those patients in our clinical trials and that drug supply will not hinder us from completing the clinical studies and obtaining regulatory approvals that will lead to wider patient access. As such, CStone does not provide any Expanded Access programs for sugemalimab outside of clinical trials at this time. Please visit https://clinicaltrials.gov and search by company, disease or medicine for applicable clinical trials.
Consistent with the 21st Century Cures Act, CStone may revise this policy at any time.
Curadel Surgical Innovations, Inc.
Expected Application Timeframe
N/AAvailable Therapies via Single-Patient EA
At Curadel Surgical Innovations, Inc. we aspire to improve human surgery by visually enhancing anatomy of every kind with FLARE® drugs. The technology we’ve developed uses low levels of invisible near-infrared (NIR) fluorescent light and NIR cameras available in most hospitals around the world to highlight any structure in the body that could help the surgeon perform better surgery. We are currently conducting clinical trials on our products, aiming to obtain regulatory approval to make this technology available to all patients as quickly as possible. As such, we believe that participation in our clinical trials is the most appropriate way to access FLARE® drugs at this time. Information about our clinical trials, including eligibility criteria and locations, is available at https://www.clinicaltrials.gov.
Curadel Surgical Innovations, Inc. understands the interest of patients and surgeons in accessing FLARE® drugs outside of clinical trials and prior to regulatory approval, however, we do not currently offer any Expanded Access Programs.
Daiichi Sankyo
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
At Daiichi Sankyo, we recognize that there are instances when a patient has a serious or life-threatening disease or condition, for which all currently available treatment options have been exhausted and enrollment into a clinical trial is not possible. In these cases, in particular, a treating physician can request the use of an investigational Daiichi Sankyo product prior to regulatory approval for the particular condition or indication, provided it is allowed by the applicable local laws.
Daiichi Sankyo strives for an equitable balance between the public interest in securing the approval of a new drug and allowing access to certain investigational medicinal products that may have the potential to treat seriously ill patients who cannot be satisfactorily treated with commercially available products. The following criteria are typically required before Daiichi Sankyo would consider patients eligible for expanded access to a Daiichi Sankyo investigational product. All criteria are subject to local laws and regulations:
--The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
--The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
--The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
--The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive.
--Daiichi Sankyo has decided to seek marketing approval in at least one major market globally.
--The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
--The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.
Daiichi Sankyo cannot approve expanded access use requests in cases where doing so would violate company policy, local laws or regulations, or if access would present an unacceptable benefit/risk profile, as deemed by medical personnel of the company. We commit to a careful and fair evaluation of each request by the appropriate medical experts at Daiichi Sankyo within the boundaries of local laws and regulations.
Available Therapies via Single-Patient EA
Daiichi Sankyo does not currently have formal expanded access programs for our other investigational products. We consider granting expanded access to products other than quizartinib on a case-by-case basis, as long as such provision will not delay, interfere with or compromise the completion of clinical trials that are intended to support approval by regulatory authorities, which, in turn, provides access to the medication for the greatest number of individuals.
Disease/Category-Specific EA Policies/Criteria
DBV Technologies, Inc.
Expected Application Timeframe
1 business dayDiakonos Oncology Corporation
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
Diakonos Oncology Inc. is a biotechnology company dedicated to the development of immunotherapies to treat late-stage cancers through a novel approach to Dendritic Cell Vaccines. DOC1021 is a multi-step process that harnesses the patient’s natural immune response, utilizing the manner in which the body mounts an anti-viral response to target and eliminate cancer cells.
Currently, access to our Dendritic Cell Vaccine can be obtained through enrollment in one of our clinical studies for Glioblastoma Multiforme (NCT04552886) and Pancreatic Adenocarcinoma (NCT04157127). The objective of these clinical studies is to evaluate patient safety and efficacy
For expanded access use to our investigational products, we encourage patients to contact us at expandedaccess@diakonosoncology.com for an evaluation.
Diakonos Oncology will assess each request on a case-by-case scenario; however, we cannot guarantee that investigational products will be made available. In any requests for expanded access use, please consider the following the US Food and Drug Administration guidelines for Expanded Access Programs.
Available Therapies via Single-Patient EA
Dendritic Cell Vaccine for the treatment of individuals diagnosed with Glioblastoma Multiforme and Pancreatic Adenocarcinoma.
Direct Biologics, LLC
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
- There must be adequate supply of the investigational product to meet the needs of the expanded access program without impacting the company’s clinical trials
- There is a compelling medical and scientific rationale for the requested use
- The potential benefit to the patient must outweigh the collective potential risks
- There must be sufficient clinical data to identify an appropriate dose
- The patient’s physician has determined that treating the patient with the investigational product is in the patient’s best interests
Available Therapies via Single-Patient EA
ExoFlo (extracellular vesicles isolated from human bone marrow mesenchymal stromal/stem cells)
Dizal Pharmaceutical Co., Ltd
Expected Application Timeframe
NASingle-Patient EA Policies/Criteria
Sunvozertinib (DZD9008)
At Dizal we aspire to discover and develop differentiated therapeutics for the treatment of cancer and immunological diseases. One of the Dizal's lead products, sunvozertinib, is an oral EGFR tyrosine kinase inhibitor (TKI). It is currently being investigated as a single agent in phase 2 and phase 3 clinical trials for the treatment of Non-Small Cell Lung Cancer (NSCLC) patients with EGFR Exon20ins mutation. We are enrolling and conducting clinical trials aiming at gaining regulatory approval to make sunvozertinib available to all eligible patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access sunvozertinib at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at clinicaltrials.gov.
Dizal understands the interest of patients in accessing drugs, such as sunvozertinib, outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases. Because sunvozertinib is an investigational drug, and its efficacy and safety are still under evaluation, sunvozertinib can only be administered in accordance with local investigational drug requirement by an experienced physician.
Dizal now offers Individual Patient Access for its investigational drug sunvozertinib for the treatment of adult patients with Advanced Non-Small Cell Lung Cancer (NSCLC), with EGFR or HER2 mutation, at the sites participating in ongoing clinical trials of sunvozertinib. Individual patients at these sites may be able to access sunvozertinib if they are ineligible for the available standard of care and are recommended by the treating physician to the Individual Patient Access Program.
In order to meet FDA requirements, Dizal will provide a Letter of Authorization (LOA), referencing the content in Dizal’s US IND, to each treating physician to support their written requests (IND) for individual patient use. This same principle may be applied to countries with IND/CTA of sunvozertinib opened to fulfill the local regulatory requirements.
Golidocitinib (DZD4205, AZD4205)
At Dizal, we aspire to discover and develop differentiated therapeutics for the treatment of cancer and immunological diseases. One of Dizal's lead products, golidocitinib, is an oral, potent, highly selective Janus kinase 1 (JAK1) inhibitor. It is currently being investigated as a single agent in a phase 2 clinical trial for the treatment of patients with relapsed/refractory peripheral T-cell lymphoma (r/r PTCL). We are conducting clinical trials aiming at gaining regulatory approval to make golidocitinib available to all eligible patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access golidocitinib at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at clinicaltrials.gov.
Dizal understands the interest of patients in accessing drugs, such as golidocitinib, outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases. Because golidocitinib is an investigational drug, and its efficacy and safety are still under evaluation, golidocitinib can only be administered in accordance with local investigational drug requirements by an experienced physician.
Dizal now offers Individual Patient Access to the investigational drug golidocitinib for the treatment of adult patients with r/r PTCL at the sites participating in ongoing clinical trials of golidocitinib. Individual patients at these sites may be able to access golidocitinib if they are ineligible for the available standard of care and are recommended by the treating physician to the Individual Patient Access Program.
In order to meet the FDA's requirements, Dizal will provide a Letter of Authorization (LOA), referencing the content in Dizal's US IND, to each treating physician to support their written requests (IND) for individual patient use. The same principle may be applied to countries with IND/CTA of golidocitinib opened to fulfill the local regulatory requirements.
Available Therapies via Single-Patient EA
DZD9008 (INN name: sunvozertinib): Non-Small Cell Lung Cancer (NSCLC) patients with EGFR Exon20ins mutation
DZD4205 (AZD4205, INN name: golidocitinib): Relapsed/refractory peripheral T-cell lymphoma (r/r PTCL)
Dren Bio, Inc.
Expected Application Timeframe
Average 2 weeksAvailable Therapies via Single-Patient EA
Dren Bio does not currently offer expanded access to our investigational products. Dren Bio believes that the most appropriate access to our investigational products is through participation in any of our ongoing clinical trials because this offers the best means to ensure the safe product usage and to generate the necessary data for product approval by regulatory agencies.
Egetis Therapeutics, Inc.
Single-Patient EA Policies/Criteria
The goal of this program is to provide expanded access (i.e., before marketing authorization) to tiratricol as treatment for patients with monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome [AHDS]), who in their Treating Physician's opinion, could benefit from tiratricol and meet the eligibility criteria.
Available Therapies via Single-Patient EA
Therapy: tiratricol - currently and investigational drug for the treatment of monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome [AHDS].
Eisai
Expected Application Timeframe
2 business daysEli Lilly
Expected Application Timeframe
5 business daysEllipses Pharma
Expected Application Timeframe
3 Business DaysSingle-Patient EA Policies/Criteria
Last updated: February 2024
Ellipses Pharma Limited is a clinical-stage precision drug development biotechnology company focused on developing novel therapies for cancers. We conduct clinical trials to evaluate the safety and efficacy of investigational product candidates.
Ellipses encourages awareness of, and participation in, our clinical trials. For information regarding these trials, please visit clinicaltrials.gov and search for Ellipses Pharma.
Our current focus is to complete our ongoing clinical trial for EP0031 to demonstrate safety and efficacy and to obtain regulatory approval and provide widespread availability of EP0031.
Prior to regulatory approval by global regulatory authorities, patients gain access to investigational treatments, including Ellipses’ development candidates, by participating in clinical trials. In certain cases, when it is not possible for a patient to participate in a clinical trial, and where all other available medical options have been exhausted, a patient’s doctor may attempt to seek special access to an investigational product candidate for that patient outside of a clinical trial. This is known, among other terms, as expanded access or compassionate use.
For a patient to obtain access to an investigational product candidate through expanded access, the patient’s doctor, the drug’s sponsor and the regulatory authority in the doctor’s country of practice (e.g., FDA in the US or the MHRA in the UK etc) must all approve the use. Unlike the use of an investigational new drug in a clinical trial setting, the primary purpose of expanded access is to use the investigational drug for patient treatment purposes, rather than to gather data on safety, tolerability and effectiveness.
Currently, Ellipses Pharma does not offer an expanded access program. We believe that access to our investigational product candidates should be limited to controlled clinical trials until such time as their safety, tolerability and effectiveness have been determined and confirmed by regulatory authorities.
Additionally access to our development candidates before dose and dosing regimen have been optimized and safety and tolerability have been confirmed may not be in the best interest of patients.
Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials are the only safe and appropriate way to access our investigational product candidates.
In accordance with the 21st Century Cures Act this policy may be revised at any time and will be shared with the public on the Company website (ellipses.life) and the Reagan-Udall Foundation Expanded Access Navigator (navigator.reaganudall.org)
Should Ellipses Pharma decide to make its investigational medicinal products available on an expanded access basis, this policy will be updated with a hyperlink to the expanded access record on clinicaltrials.gov after that record becomes active.
If you have questions about Ellipses Pharma’s expanded access policy, please contact us at expanded.access@ellipses.life.
Ellipses Pharma Limited may revise this policy at any time.
Available Therapies via Single-Patient EA
Currently, Ellipses Pharma does not offer an expanded access program. We believe that access to our investigational product candidates should be limited to controlled clinical trials until such time as their safety, tolerability and effectiveness have been determined and confirmed by regulatory authorities.
EMD Serono
Expected Application Timeframe
5 business daysEntasis Therapeutics
Expected Application Timeframe
3 business daysEpirium Bio Inc
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Epirium Bio is committed to developing safe and effective therapies for intractable neuromuscular and neurodegenerative diseases associated with mitochondrial depletion, as well as primary mitochondrial disorders, and providing those therapies to the broadest group of patients as quickly as possible. We also recognize that there are many diverse conditions in which mitochondrial depletion or dysfunction are a key component of the disease process. As part of our commitment to the rare disease community, we will support compassionate use / expanded access programs* when we have substantial scientific evidence to support both the safety and the potential efficacy of an investigational medical product for a given indication, and when it is logistically practicable.
Epirium has a developed a process for determining whether the company will provide an experimental therapy under compassionate use.
In the first step, the company will evaluate whether:
• there is substantial scientific evidence to support both the safety and the efficacy of an investigational medical product for a particular indication;
• it has been established that access on a compassionate use basis will not compromise clinical trials or the regulatory pathway for an investigational medical product;
• there is adequate supply of the investigational medical product; and
• the investigational medical product can be administered – and it is logistically feasible to make it available – outside of the clinical trial setting.
If the company decides that, under the first step, availability of the investigational medical product on a compassionate use basis is possible, then the company will evaluate an individual’s request for access.
This second step uses the following criteria:
• the patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition;
• there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
• patient enrollment in a clinical trial is not possible;
• potential patient benefit justifies the potential risks of treatment;
• providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication, and;
• all necessary regulatory/institutional approvals have been obtained to allow the administration of the investigational medical product.
Requests for access to investigational medical products must be made by a qualified and licensed physician and will be evaluated by Epirium. Patients interested in seeking expanded access to an Epirium investigational medical product should talk to their physician. Qualified and licensed physicians may make compassionate use / expanded access requests by contacting Epirium by e-mail at info@epirium.com. Epirium anticipates it will acknowledge receipt of such requests within five business days of their receipt
* Compassionate use programs include requests for access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, as well as ‘Right to Try’ legislation.
Available Therapies via Single-Patient EA
EPM-01 ((+)-epicatechin)
Disease/Category-Specific EA Policies/Criteria
https://epirium.com/wp-content/uploads/2020/05/Epirium_Bio_Statement_On_Compassionate_Use.pdf
Erytech
Expected Application Timeframe
48 hoursSingle-Patient EA Policies/Criteria
Erytech’s Public Policy – Compassionate Use (Expanded Access)
Erytech has no marketing approvals for any of its products throughout the globe.
Erytech is a science-led organization researching and developing new medicines through its ERYCAPS® platform. We are currently operating in North America and Europe.
This policy sets out the general principles for Erytech providing compassionate use. It is intended as global guidance, although Erytech will act in strict accordance with all local country laws and regulations on providing compassionate use.
Erytech’s lead product, eryaspase, is supported by clinical evidence from late stage clinical development1.
Erytech is currently conducting late stage clinical trials in second-line pancreatic cancer (Trybeca-1) in Europe and the US, and in first-line Triple Negative Breast Cancer (Trybeca-2) in Europe. More details, including participating investigator centres, can be found on CT.gov or by sending an email to:
medaffairs@erytech.com
Erytech recognises that not all cancer patients will be suitable candidates for these studies. Where enrolment into a clinical trial is not an option for them, it may be in the interest of patients to have access to eryaspase. Under these circumstances, and where patients are in a life-threatening situation with no satisfactory alternative treatment options, Erytech may provide a treating healthcare professional (HCP) with an investigational medicine such as eryaspase.
As general guidance, Erytech will consider providing Compassionate Use according to the following:
• Erytech considers the appropriateness of compassionate use for all our investigational medicines early on in the planning of our research programmes. At an early stage in a medicine’s development there could be only limited understanding. For example:
- Uncertainty on the best way to provide the medicine to patients, such as the exact dose to use and frequency.
- Establishing the medicine’s efficacy and safety profile.
- Erytech provides compassionate use in two ways:
- Formal programmes
- Patient populations meeting specific criteria managed under a compassionate use protocol.
- Named (or individual) patients where appropriate and subject to fulfillment of our criteria.
All requests for compassionate use, which must be made by an HCP, will be considered by Erytech and according to the following:
o Patients are in a life-threatening situation.
o There are no satisfactory alternative treatments (confirmed by the HCP).
o There is sufficient evidence to believe the potential benefit to the patient justifies the risk.
• Compassionate use decisions are made by Erytech’s Chief Medical Officer and are final.
Other key considerations include:
• Any concern that compassionate use of the investigational medicine might somehow compromise any related clinical trial or regulatory pathway.
• Restricted to countries where Erytech is able to provide its product. Currently, this is North America and Europe.
• That use in formal progammes will only take place in countries where Erytech intends to seek regulatory approval and to make the medicine available; the same limitation will not necessarily apply for named (or individual) patients.
• That the proposed compassionate use complies with local laws and regulations.
These criteria ensure Erytech has a consistent approach for our decisions on compassionate use.
To investigate further options for the appropriate access to Erytech’s medicines, any HCP can contact Erytech by sending an email to:
medaffairs@erytech.com
Erytech will acknowledge receipt of a compassionate use request submitted by an HCP within 48 hours.
Patients interested in accessing an Erytech medicine for compassionate use should talk to their doctors.
Erytech is committed to transparency in its interactions with HCPs and health care organizations/institutions consistent with applicable laws and/or codes of practice applicable to the pharmaceutical industry. Erytech will fulfil all regulatory requirements to make public information about our compassionate use activities.
1. Hammel P, Fabienne P, Mineur L, et al. Erythrocyte-encapsulated asparaginase (eryaspase) combined with chemotherapy in second-line treatment of advanced pancreatic cancer: An open-label, randomized Phase IIb trial. Eur J Cancer. 2019;124:91- 101.
Available Therapies via Single-Patient EA
Eryaspase
Currently under clinical study in:
- Pancreatic cancer
- Triple Negative Breast Cancer
- Acute Lymphoblastic Leukaemia (ALL)
Disease/Category-Specific EA Policies/Criteria
Esperare
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
ER-004 COMPASSIONATE USE/EXPANDED ACCESS POLICY in XLHED
Expanded access/Compassionate use refers to the use of an investigational therapy, that have not yet been approved by government regulatory agencies, outside a clinical trial, for patients with serious or life- threatening diseases or conditions who lack therapeutic alternatives.
Esperare’s mission is to advance treatments for rare diseases and to promote treatment accessibility through a patient-centered approach.
Accordingly, a Patient Advisory Council dedicated to the XLHED program has been established and provides the opportunity to Esperare and patient groups representatives to work in close collaboration. In particular, clinical development plans are discussed in order to refine development and generate robust and necessary data for an application and to contribute to the common goal of giving the greatest chance of success to this program, with the ultimate objective of maximizing the benefits and minimizing the risks for XLHED patients and their families.
Requests for compassionate use/expanded access will be considered on a case by case basis , in accordance with applicable regulations and in alignment with our mission and values and with the safety of the patient and childbearing mother as a priority. Of note, ER-004 is delivered to the patient before birth by injection into the amniotic fluid, an untried route of administration that requires careful consideration in each individual case.
Inquiries regarding the expanded access may be sent to info.er004@esperare.org.
Esperare will endeavor to acknowledge receipt of any expanded access questions or requests within 5 business days.
Available Therapies via Single-Patient EA
ER-004 (also known as EDI200) is a protein replacement therapy designed as a substitute for endogenous EDA, a protein missing in XLHED.
Exelixis, Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Exelixis believes that the best way for a patient to have access to an investigational medication is through a clinical trial. Clinical trials, which may be sponsored by Exelixis or by another institution such as an academic medical center or a governmental body, enable rigorous systematic evaluation of a medication and allow for close monitoring of enrolled patients. Clinical trials, including eligibility criteria, are listed at ClinicalTrials.gov. Patients interested in learning if enrollment in a clinical trial is an option for them should consult with their treating physician.
Patients who are not eligible for enrollment in a clinical trial may be able to access an investigational medication via early access. Early access is only available in limited circumstances for certain types of patients for which no other treatment options exist. Provision of an investigational medication by Exelixis will always be done in accordance with applicable laws and regulations. At Exelixis, early access to an investigational medication outside of a clinical trial might be possible through an “Expanded Access Program,” a single-patient investigational new drug application submitted to the U.S. Food and Drug Administration, or via a “Named Patient Use” program in other countries.
The following are some of the general considerations for early access:
- For an Exelixis investigational medication to be made available to a patient via early access, the patient must be diagnosed with a serious or immediately life-threatening disease for which no satisfactory treatment alternative exists.
- The medication must be under clinical development by Exelixis, and the provision of the medication must not compromise the clinical development program (e.g., the initiation, conduct or completion of trials) or the potential regulatory approval of the medication.
- Exelixis must have adequate supply of the medication.
- Sufficient data must exist to support its use in the specific clinical condition from which the patient suffers, and adequate data must also exist to identify an appropriate dose for use in such patients.
- Overall, the potential benefit to the patient must outweigh the potential risks to the patient.
Patients who are interested in obtaining early access to an Exelixis investigational medication should discuss the matter with their treating physician. A request for early access must come directly from the physician and should be conveyed to Exelixis Medical Information at medinfo@exelixis.com or by calling 1-855-292-3935. Physicians who administer Exelixis investigational medications through early access are required to comply with all applicable laws and regulations and contractual conditions, including those relating to safety reporting.
Faron Pharmaceuticals Ltd
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
General Policy
Faron Pharmaceuticals does not have an open Expanded Access program at this time. Participation in one of our ongoing clinical trials is the most appropriate way to access bexmarilimab and other investigational therapies. We encourage patients to speak with their physicians about the possibility of enrolling in a clinical trial listed on ClinicalTrials.gov.
Revisions to the Policy
Consistent with the 21st Century Cures Act, Faron Pharmaceuticals may revise this policy at any time without prior notice. Updates to the policy will be made available on this directory, our website and through appropriate communication channels.
Forte Biosciences, Inc.
Phone Number & Email
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
Expanded access, also called “compassionate use”, enables some patients with an immediately life-threatening condition or serious disease to gain access to an investigational new drug for treatment outside of clinical trials when no alternative therapy options are available. Currently, Forte does not offer an expanded access program. Forte may revise this policy at any time.
Disease/Category-Specific EA Policies/Criteria
Frontier Biotechnologies Inc.
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
The IND has been deemed safe-to-proceed and the fast track designation has been granted. The registration in ClinicalTrials.gov will be done before the trial starts and a link will be provided then.
FUJIFILM Pharmaceuticals U.S.A., Inc.
Single-Patient EA Policies/Criteria
Currently, FUJIFILM Pharmaceuticals U.S.A., Inc. is not accepting expanded access/compassionate use requests.
Gan & Lee Pharmaceuticals
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
An Expanded Access Program provides HCPs and patients access to investigational therapies when the option of enrolling in a clinical trial may not be available. These programs may also be referred to as Compassionate Use Programs.
Currently, Gan & Lee is not offering an Expanded Access Program. To learn more about available clinical trials by Gan & Lee, please visit http://clinicaltrials.gov and search by company, disease, or compound.
Please feel free to contact us with any questions you may have.
Gannex Pharma Co., Ltd.
Expected Application Timeframe
15 business daysSingle-Patient EA Policies/Criteria
Gannex Pharma Co.,Ltd. (Gannex), a wholly-owned company of Ascletis, is fully dedicated to the R&D and commercialization of drugs in the field of Non-alcoholic steatohepatitis(NASH). Gannex conducts clinical trials in individuals to evaluate investigational medicines in order to obtain information on safety and efficacy that may be used to support marketing approval and subsequent wider accessibility of the product to patients. Investigational medicines are drugs that have not been approved by regulatory authorities.
Gannex has three clinical stage drug candidates against three different NASH targets Fatty Acid Synthase(FASN), Thyroid Hormone Receptor-Beta(THR-β) and Farnesoid X Receptor(FXR), and three pre-clinical stage NASH combination therapies. Gannex seeks to retain the ability to manufacture and supply investigational medicines in a fair and equitable manner and in a volume that assures adequate supply for ongoing clinical trials and development programs. Hence, at this time, Gannex is unable to provide its investigational medicines on an expanded access or right to try basis. For patients seeking access to our investigational medicines before they are approved by a regulatory authority, participation in one of Gannex’s clinical trials is the most appropriate way to access these investigational medicines. To learn more about available clinical trials by Gannex, please visit www.gannexpharma.com, or visit https://clinicaltrials.gov and search by company, disease or medicine.
If you have additional questions, please speak with your physician or contact Gannex at ea@gannexpharma.com.
Consistent with the 21st Century Cures Act, Gannex may revise this policy at any time.
Disease/Category-Specific EA Policies/Criteria
GC Biopharma
Expected Application Timeframe
5 Business DaysAvailable Therapies via Single-Patient EA
Expanded Access Policy for Sanfilippo Syndrome Type A
GC Biopharma is currently developing GC1130A, a potential enzyme replacement therapy for patients with Sanfilippo syndrome type A. We understand there may be interest in access to investigational product for patients outside of clinical trial. At this stage of clinical development, participation in a clinical study is the only way to gain access to GC Biopharma’s investigational product.
Genentech
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
https://www.gene.com/patients/clinical-trial-information/other-types-of-access#general-criteria
GenSight Biologics
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
GENSIGHT BIOLOGICS POLICY ON EXPANDED ACCESS TO INVESTIGATIONAL THERAPIES
Investigational therapy covered by this Policy
GS030 combination product for the treatment of Retinitis Pigmentosa
About Expanded Access
GenSight Biologics is focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Clinical trials form the foundation of our development programs. These trials are designed to assess the safety and efficacy of investigational medicines. Participation in a clinical trial represents the best way, in a controlled setting, to gain access to an investigational medication. Information about ongoing GenSightsponsored clinical trials can be accessed by consulting https://clinicaltrials.gov/. In some circumstances, a patient may not be able to participate in a clinical trial. Seeking use of an investigational medication under these circumstances is permitted by the FDA and commonly referred to as compassionate use or expanded access. Our policy is intended to comply with U.S. Food and Drug Administration (FDA) requirements for any such use or access.
At this time, given the early stage of development, we are not making our investigational therapy, GS030 combination product, available on an expanded access or compassionate use basis anywhere in the world. Generally, the safety and efficacy data needed to grant expanded access are not available until the initiation of phase 3 clinical trials. For now, participation in our phase 1/2 clinical trial, called PIONEER, is the only way to receive our investigational therapy.
If you are interested in participating in GenSight Biologics clinical trial and would like more information, or if you have any questions about this expanded access policy, please talk to your doctor or contact us https://www.gensight-biologics.com/form/contact-us/.
We will continue to evaluate the possibility of expanded access as we advance development of GS030 combination product. As authorised by the 21st Century Cures Act, GenSight Biologics may revise this posted expanded access policy at any time.
The availability of this policy or any revised version shall not serve as a guarantee of access to GS030 by any individual patient.
More information about GenSight Biologics’ ongoing clinical trial for Retinitis Pigmentosa, PIONEER, can be found by visiting the link: https://clinicaltrials.gov/ct2/show/NCT03326336.
12 October 2021
Available Therapies via Single-Patient EA
At this time, none of our therapies are available via single-patient EA.
Disease/Category-Specific EA Policies/Criteria
GlaxoSmithKline
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
http://www.gsk.com/media/3368/compassionate-use.pdf
Go to GSK Compassionate Use Request Portal at https://gsk-cu-portal.idea-point.com/ to make a request.
GlobalMed Technologies USA,Inc
Expected Application Timeframe
24-48 hoursSingle-Patient EA Policies/Criteria
GMTUSA development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational device treatment, and to obtain regulatory approval.
GMTUSA is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to GMTUSA's investigational PCI-01 device treatment by contacting the Company.
The purpose of this policy is to describe the requirements for Expanded Access to GMTUSA investigational PCI-01 device treatment to patients outside of a clinical study.
Scope
This policy applies to provision of access to a GMTUSA investigational device product that is not approved for any purpose in the country from which the request is intended to be used. This also includes the time period between regulatory approval of an investigational product and its commercial availability in a country.
Policy Statements
Any use of a GMTUSA investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including COGMTUSA policies and procedures.
In general, where permitted by local regulation, the investigational product supplied via Expanded Access may no longer be provided by GMTUSA when it becomes available via the local healthcare system.
GMTUSA may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the country.
A. Patient Eligibility Criteria
To be eligible for access to an investigational product, patients must meet the
following criteria:
• Suffer from a serious or immediately life-threatening disease or condition (COVID-19).
• Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• The patient has a disease (COVID-19) for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• There is adequate information to support appropriate dosing for a special population patients such as pediatric, elderly, renal or hepatic disease, etc.
•Any other pertinent medical criteria for access to the investigational product, as established by the GMTUSA clinically or medically responsible individual.
B. Investigational Product Criteria
In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
• The product is under investigation in one or more clinical studies.
• There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
• The provision of the investigational product will not interfere with or compromise the clinical development of the product.
C. Treating Physician Criteria and Responsibilities
The physician(s) attending to the patient(s) who is/are receiving an investigational product through compassionate use access is (are) properly licensed and fully qualified to administer the product. The physician must agree in writing to comply with:
• Any applicable country-specific legal and regulatory requirements related to providing an investigational product under Expanded Access.
• Any CGMTUSA requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property. A treating physician may submit questions or requests regarding expanded access to drfernandez@gmtpci.com
GlycoMimetics, Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
GlycoMimetics considers expanded access requests on a case-by-case basis. Consideration factors include: the patient’s ability and eligibility to participate in an ongoing trial, drug supply and availability, the treating physician’s benefit-to-risk analysis, and institutional experience with the drug, among others.
Available Therapies via Single-Patient EA
Uproleselan for use in combination with chemotherapy for treatment of adult patients with relapsed or refractory acute myeloid leukemia.
Gyroscope Therapeutics
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Gyroscope Therapeutics’ mission is to preserve people’s sight and fight the devastating impact of blindness around the world. We embarked on this journey because each year millions of people lose their vision due to diseases that we believe may one day be treatable. Our current focus is to discover and develop pioneering gene therapies for one of the leading causes of vision loss – age-related macular degeneration (AMD).
To realise this mission, it is our duty to conduct the research and clinical trials necessary to evaluate the safety and effectiveness of our investigational medicines. We believe that rigorous clinical trials are the most appropriate way to do this. We are committed to working closely with researchers, doctors, clinical trial site teams, patients and families to enrol, conduct and analyse our studies. If successful, data from these studies may support submissions to regulatory authorities (such as the FDA and EMA) for potential approval – with the ultimate goal of making our medicines available to as many eligible patients in need as safely and quickly as possible.
At this time, we are not making our investigational gene therapy, GT005, available on an expanded access basis anywhere in the world (expanded access is sometimes also referred to as compassionate use or pre-approval access). For now, participation in one of our clinical trials is the only way to receive our investigational gene therapy. This decision was made after careful evaluation of many factors, including: the safety and effectiveness of GT005 is still being evaluated in early stage clinical trials; our manufacturing capacity for GT005; considerations related to one-time gene therapies; and, what we believe is in the best interest of patients.
We understand there are currently no approved treatments for dry AMD, which is why we are working hard to develop our gene therapies as quickly as we can. There are many clinical trials evaluating potential new therapies for this devastating condition. We recommend that you talk to your doctor about what is right for you.
If you are interested in participating in one of Gyroscope’s clinical trials and would like more information, or if you have any questions about this expanded access policy, please talk to your doctor or contact us. We anticipate acknowledging receipt of expanded access questions or requests sent to us within five (5) business days of receipt.
More information about Gyroscope’s ongoing clinical trials can be found by visiting the links below.
We will continue to evaluate the possibility of expanded access as we advance development of our investigational medicines. As authorised by the 21st Century Cures Act, Gyroscope may revise this posted expanded access policy at any time.
The availability of this policy or any revised version shall not serve as a guarantee of access to GT005 or any other investigational medicines by any individual patient.
Current trials of GT005 in geographic atrophy secondary to dry AMD:
Available Therapies via Single-Patient EA
At this time, none of our therapies are available via single-patient EA.
Disease/Category-Specific EA Policies/Criteria
Hanmi Pharmaceutical Co., Ltd
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
Any use of Hanmi’s investigational products outside clinical trials should be made in accordance with local regulations and guidelines as well as Hanmi’s procedures and policies.
Hanmi will not grant access to Hanmi’s investigational products if the expanded access will interfere with the company’s ability to complete clinical trials as planned or will delay the drug development program.
Hanmi will grant access to Hanmi’s investigational products when there is an adequate supply of the investigational products to meet the needs of the expanded access without impairing clinical trials for drug development.
Hanmi will provide investigational product with 3 months’ treatment supply per case. In case treating physician judges that it is beneficial for patients to continue Hanmi’s investigational products, new apply should be made.
In general, where permitted by local regulation, an investigational product provided via expanded access according to local regulations and guidelines will no longer be provided by Hanmi once it becomes available on the market.
Hanmi may decide not to provide an investigational product under this policy if Hanmi decides to stop the drug development or not to launch in the concerned country(ies).
Available Therapies via Single-Patient EA
LAPS-Triple Agonist (also known as HM15211) is a long-acting Glucagon/GIP/GLP-1 triple agonist chemically conjugated with constant region of human immunoglobulin via non-peptidyl flexible linker. The therapeutic indication is non-alcoholic steatohepatitis (NASH).
LAPSGLP-2 Analog (also known as HM15912) is a long-acting glucagon-like peptide-2 (GLP-2) analogue linked to a human immunoglobulin G4 fragment crystallizable. The therapeutic indication is short bowel syndrome in the pediatric and adult populations.
Disease/Category-Specific EA Policies/Criteria
http://www.hanmipharm.com/ehanmi/handler/Rnd-FocusedPipelineA
http://www.hanmipharm.com/ehanmi/handler/Rnd-FocusedPipelineB
HUTCHMED Limited
Expected Application Timeframe
3 business daysILiAD Biotechnologies
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
ILiAD is committed to developing safe and effective vaccines, with a goal to providing our next generation vaccines as soon as possible through efficient vaccine development. At this time, ILiAD’s vaccines are in the investigational phase and not yet marketed. ILiAD does not have an expanded access program.
Disease/Category-Specific EA Policies/Criteria
Imaging Biometrics, LLC
Expected Application Timeframe
1 DaySingle-Patient EA Policies/Criteria
We are considering an intermediate group EAP.
Imara, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Imara is dedicated to developing novel therapeutics for patients with sickle cell disease and other hemoglobinopathies. Sickle cell disease represents a critical unmet medical need globally in which it is considered as a rare disease in many parts of the world, including in the United States, and as an endemic condition in several African countries. Imara’s lead product candidate, IMR-687, is under development for the treatment of sickle cell disease.
Expanded access may provide an avenue to use an investigational product, such as IMR-687, outside a clinical trial to diagnose, monitor, or treat a serious condition or disease in a patient. Imara has used guidelines from the US Food and Drug Administration (US FDA) and other regulatory agencies to develop the following criteria for when expanded access may be made available on a case-by-case basis for individual patients:
• Ongoing or planned clinical studies are not available to the patient, including lack of access due to geographical location of potential clinical trial sites,
• Significant evidence exists that supports both the safety and the efficacy of the investigational drug for the indication,
• The potential benefits to the particular patient seeking access to the investigational drug outweigh the potential risks to the patient,
• An adequate supply of the investigational drug exists,
• All necessary regulatory/institutional approvals have been obtained to allow drug administration, and
• The request for expanded access has been made by a qualified health care provider with expertise appropriate for the administration of the drug and for monitoring and managing the patient.
Imara supports expanded access programs and the need for a suitable policy, and it intends to provide patients with sickle cell disease access to IMR-687 at a suitable time and in the correct method when used outside a clinical trial. At this time, Imara believes that the most appropriate way to use our investigational lead product candidate, IMR-687, is by participation in one of our clinical trials (https://clinicaltrials.gov/).
Available Therapies via Single-Patient EA
IMR-687 for sickle cell disease
Immix Biopharma
Expected Application Timeframe
24hr to 48hrSingle-Patient EA Policies/Criteria
http://immixbio.com/pipeline/compassionate-use-policy/
Available Therapies via Single-Patient EA
imx-110 - all advanced solid tumors
ImmuneOnco Biopharmaceuticals (Shanghai) Inc.
Expected Application Timeframe
7 business daysSingle-Patient EA Policies/Criteria
Immuneonco will consider providing a requesting physician with pre-approval access to a specific investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:
- The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option.
- The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition.
- A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available.
- Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access.
- Adequate supply of the investigational drug is available.
Immuneonco is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician. Immuneonco may require more detailed information in order to fully evaluate a request. Each request will be given careful consideration by Immuneonco whose decisions are final.
Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to the mailbox (info@immuneonco.com). We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 7 working days after receipt.
To learn more about the EA policy by Immuneonco, please visit www.immuneonco.com.
Available Therapies via Single-Patient EA
IMM2902
IMM2902 is a recombinant bispecific monoclonal antibody. The indication is the use of IMM2902 in patients with HER2-expressing advanced solid tumors, breast cancer and gastric cancer.
Disease/Category-Specific EA Policies/Criteria
Immunic
Single-Patient EA Policies/Criteria
Immunic is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient
communities through education, empathy, and awareness. Consistent with Immunic’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who
participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing. At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Immunic will consider providing a requesting physician with pre-approval access to a specific Immunic investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.
These conditions include the following:
- The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
- The investigational drug is in active clinical development, with the clinical development having reached the stage of blinded Phase 3 data read-out
- A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
- Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
- Adequate supply of the investigational drug is available.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.
Immunic is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Immunic may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Immunic whose decisions are final.
Immunomedics
Expected Application Timeframe
5 business daysImmvira Pharma Co. Ltd
Expected Application Timeframe
10 Business DaysSingle-Patient EA Policies/Criteria
ImmVira is a biotechnology company focused on development of new generation novel drug vectors driven by clinical benefits in oncology and non-oncology fields. T3011 is a replication-competent, genetically modified virus derived from herpes simplex virus type 1 expressing IL-12 and PD-1 antibody.
ImmVira recognizes that some patients with serious or life-threatening disease may lack other treatment options and be unable to participate in a clinical trial. Under these circumstances, a healthcare professional (HCP) may request access to ImmVira's investigational medicines for patient treatment. This access is called "compassionate use" or "expanded access" and allows the use of an unapproved drug outside of a clinical trial.
This policy outlines ImmVira's process for reviewing requests from HCP for compassionate use of our investigational drugs. To be considered, the patient's condition must be:
• Severity of the patient's condition: The illness must be life-threatening or significantly impact daily life.
• Lack of alternatives: The doctor treating the patient must confirm there are no other suitable treatments available.
• Potential benefit outweighs risk: There must be evidence suggesting the potential benefits of the investigational drug are greater than the potential risks for this specific patient.
Beyond the initial criteria, several factors influence our decision on supplying investigational drugs:
• Treatment Guidance: Clear instructions for dosage and treatment duration are necessary for safe use.
• Clinical Trials: We evaluate if access might affect ongoing clinical trials or the drug's regulatory path.
• Medical Infrastructure: The treatment can only be provided in countries with facilities to safely administer it.
• Broad Access Programs: We prioritize countries where we plan to seek approval and make the drug commercially available. This might not apply to individual patients.
• Ethical Considerations: We ensure fair treatment principles are followed for all patients.
• Legal Compliance: The treatment must comply with local regulations.
• Drug Availability: We must have sufficient supply of the investigational drug to fulfill the request.
We strongly believe that participation in one of our clinical trials is the most effective way to access T3011.
ImmVira will also consider providing a requesting physician with pre-approval access to T3011, for the treatment of an individual patient outside of a clinical trial. ImmVira is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s physician. Each request will be given careful consideration by ImmVira whose decisions are final.
HCPs seeking to request unlicensed medicines for treatment use should file the treatment IND to FDA and submit relevant information to IRB.
For further information on submitting a request for treatment use of our investigational medicines, HCPs are directed to follow this link https://www.immviragroup.com/.
Available Therapies via Single-Patient EA
T3011 Intratumoral Injection in Patients With Advanced Solid Tumors
Indapta Therapeutics, Inc.
Expected Application Timeframe
2 business daysSingle-Patient EA Policies/Criteria
Indapta’s Expanded Access Policy (EAP) covers the use of its investigational products outside of an ongoing clinical trial. Indapta appreciates the need for additional treatments for patients who have serious or immediately life-threatening diseases and have limited available treatment options. The decision to establish an EAP is dependent on a number of key factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ requirements. These include, but are not limited, to the following:
• The illness for which the investigational cell therapy is being considered must be serious or life-threating, with no other satisfactory treatment options (such as approved products or enrolling clinical trials);
• There is sufficient evidence that the potential benefit of the investigational drug to the patient would outweigh the potential risks, based on all available safety and efficacy information; and
• Providing the investigational cellular immunotherapy as part of an EAP will not interfere with the enrollment of clinical trials to support the development and marketing approval. Based on these factors and the early stage of clinical development for our products, Indapta believes that participation in our clinical trials is the appropriate means to access our investigational cellular immunotherapies.
Therefore, Indapta does not currently have an expanded access program. If you are a patient seeking access to our investigational product(s), please contact your treating healthcare provider.
For more information about our ongoing clinical trials, please refer to www.clinicaltrials.gov, study number NCT06119685. Healthcare providers who are interested in learning more about clinical program(s) and investigational product(s) and/or participation in our clinical trial(s), please contact trials@indapta.com.
Available Therapies via Single-Patient EA
Universal, allogeneic Natural Killer cell therapy
Indications under investigation:
-Relapsed/Refractory Multiple Myeloma
-Relapsed/Refractory Non-Hodgkins Lymphoma
Intensity Therapeutics, Inc.
Expected Application Timeframe
1 weekSingle-Patient EA Policies/Criteria
Intensity Therapeutics’ (the Company) is the developer and drug manufacturer for a novel investigational drug product, INT230-6. This new product is in early clinical development and uses intratumoral injection for its delivery modality. Recently the FDA granted Fast Track Status to INT230-6 for development in patients with triple negative breast cancer who have failed 2 lines of therapy.
The drug is being used to treat solid tumor cancers. Intensity Therapeutics has chosen not to offer EA and will not accept EA requests for its products at this time.
Available Therapies via Single-Patient EA
There are no therapies available for single-patient EA at this time.
Inventiva
Expected Application Timeframe
15 business daysInvivyd
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Currently, Invyvid does not have an expanded access program that permits access to our investigational products prior to regulatory authority (such as FDA) authorization.
InxMed
Single-Patient EA Policies/Criteria
At present we do not provide expanded access, also known as compassionate use or pre-approval access, to InxMed’s investigational medicinal products (IMP) outside the confines of clinical trials. And we do not have any expanded access programs at this time.
For information on InxMed’s available clinical trials, please visit: www.ClinicalTrials.gov or regional appropriate equivalent(s).
If you have additional questions regarding this policy, please speak with your health care provider or contact: expandedaccess@inxmed.com.
IO Biotech ApS
Single-Patient EA Policies/Criteria
IO Biotech ApS (IO Biotech) is the developer of a novel investigational drug product, IO102-IO103 Immunotherapeutic Cancer Vaccine. The investigational drug product is being evaluated for the treatment of previously untreated, unresectable or metastatic melanoma regardless of BRAF mutation status in combination with an anti-PD-1 antibody.
At the current stage of clinical development, IO Biotech has chosen not to offer Expanded Access (EA) and will not accept EA requests for the investigational drug product for the following reasons:
• IO Biotech’s ability to conduct clinical trials is limited. Offering EA under any setting at this time will impact IO Biotech’s ability to execute its trials due to a limited supply of the investigational drug product.
• IO Biotech does not have the personnel and other resources to offer EA on a fair and equitable basis.
• The investigational drug product requires special handling, transportation and training on the method of administration.
As IO Biotech is not currently accepting EA requests, the specified policy information on procedures and evaluation criteria is not provided at this time. The status of EA restriction will be re-evaluated at later development stages.
IO Biotech ApS Expanded Access Policy, Rev: 23 December 2020
Ionis Pharmaceuticals, Inc.
Expected Application Timeframe
5 Business DaysAvailable Therapies via Single-Patient EA
Olezarsen for Familial Chylomicronemia Syndrome (FCS)
At present, Ionis Pharmaceuticals has an EAP available for olezarsen (ClinicalTrials.gov ID NCT06360237). Olezarsen is an investigational antisense oligonucleotide-GalNAc3 conjugate that causes degradation of apoC-III mRNA and a reduction of serum apoC-III protein. Studies suggest that apoC-III regulates both triglyceride metabolism and hepatic clearance of chylomicrons and other triglyceride-rich lipoproteins. The expanded access program for olezarsen is intended for those at risk of severely elevated triglyceride levels, particularly those living with FCS. The program is currently only available in the United States.
If you are a patient, family member or caregiver and you wish to know more about the olezarsen EAP for FCS, please discuss the EAP and all treatment options with your treating physician.
If you are a treating physician and are seeking information about the olezarsen EAP or would like to request access for a patient, please contact patient.access@bionicalemas.com.
Bionical Emas is a Clinical Research Organization managing the olezarsen EAP on behalf of Ionis.
Safety and efficacy have not been evaluated or established by any regulatory authorities for olezarsen.
ISA Therapeutics B.V.
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
ISA Therapeutics (ISA) is developing ISA101b in HPV16 positive malignancies. The goal of our current clinical trial program is to obtain clinical data on ISA101b that is required by the US Food and Drug Administration (FDA) and other regulatory authorities for review and approval of marketing authorization applications for this investigational agent. ISA believes that focusing on clinical trials is the most appropriate way to achieve this goal; therefore, ISA101b is currently not offered outside of clinical trials and, at this time, ISA does not have an Expanded Access Program. In the event that ISA decides to consider expanded access, ISA will update its policy at that time and will then evaluate and respond to each request that it receives on a case-by-case basis. For more information on our investigational therapies and ongoing clinical trials, please visit clinicaltrials.gov.
Isofol Medical AB (publ)
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
At this point of time, we do not have an expanded access program that allows patients to have access to our investigational products prior to FDA approval.
Iterum Therapeutics plc
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Iterum Therapeutics was founded with one over-arching goal: to develop an antibiotic that addresses the growing crisis of multi-drug-resistant pathogens and meets the specific needs of physicians, their patients and other important stakeholders. Among the most important demands were new treatments for common, often serious conditions, including urinary tract infections (UTI), and intra-abdominal infections (IAI). Iterum Therapeutics is committed to conducting the clinical trials required to gain regulatory approvals to allow our medicines to be available to patients. Patients are encouraged to participate in one of our clinical trials whenever possible. For those patients with an unmet need who have exhausted all available therapy options and who are unable or ineligible to participate in a clinical trial, we may be able to provide access to our investigational medicine(s) in certain circumstances through our expanded access program.
Iterum Therapeutics may provide access for patients to our investigation medicine(s) outside of a clinical trial provided the patient meets Iterum Therapeutics’ evaluation criteria for such access. At a minimum, requests must meet the following criteria in order to be considered:
• The patient has a serious or immediately life-threatening illness when there are no comparable or satisfactory alternative treatments available and the patient is ineligible or unable to participate in a clinical trial;
• The benefit-risk analysis of the patient’s condition and medical history support the use of an investigational drug and justifies the potential risks of the treatment;
• Making the investigational therapy available for expanded access will not interfere with the initiation, conduct, or completion of clinical trials being conducted by Iterum Therapeutics that could support marketing approval for a particular treatment indication;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• There is an adequate supply of the investigational medicines;
• Provision of such access to investigational medicines is compliant with local regulations and laws.
All requests meeting the above criteria must be submitted by the patient’s treating physician to EAProgram@iterumtx.com. Iterum Therapeutics may request additional information from the patient’s treating physician to support the request. The treating physician must also agree to obtain appropriate regulatory and Institutional Review Board approvals and patient informed consent and to carry out patient monitoring and safety reporting.
Iterum Therapeutics is committed to assessing every early access request with the highest degree of fairness and equality, however, Iterum Therapeutics cannot guarantee access to any investigational medicine. Iterum Therapeutics regularly monitors this email box and will attempt to acknowledge each submitted request within 3 business days after receipt.
Available Therapies via Single-Patient EA
Sulopenem sodium (intravenous)
Sulopenem etzadroxil/probenecid (tablet)
Janssen
Expected Application Timeframe
ImmediateSingle-Patient EA Policies/Criteria
https://www.janssen.com/compassionate-use-pre-approval-access/our-policy-and-principles
Disease/Category-Specific EA Policies/Criteria
https://www.janssen.com/compassionate-use-pre-approval-access/investigational-medicines-for-compass…
Jiangsu Pacific Meinuoke Biopharmaceuticals
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
At this time, we do not have an expanded access program that allows patients to have access to our investigational products prior to completing clinical phase II study. The IND of Meplazumab for Injection (IND 143872) has been deemed partical safe-to-proceed and the fast track designation has been granted on Feb 14th, 2020. The registration in ClinicalTrials.gov will be done before the trial starts and a link will be provided then.
KalVista Pharmaceuticals Ltd
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
To serve patients, KalVista Pharmaceuticals (KalVista) engages in clinical research with the goal of obtaining regulatory approval of its products.
Clinical trials allow KalVista to evaluate new treatments in patients in order to generate the safety and efficacy information needed to obtain approval of those treatments and make them available to the broader patient population. Outside of a clinical trial, access to KalVista’s investigational products would be considered only under limited circumstances, and as permitted by applicable law, in the following situations:
- KalVista may provide continued access to its investigational products to participants in a clinical trial once the trial is complete.
- KalVista may provide physician-requested expanded access (also known as compassionate use) to its investigational products to patients who cannot join an active clinical trial of the investigational product providing the criteria below are met.
Kazia Therapeutics Limited
Expected Application Timeframe
3 business daysAvailable Therapies via Single-Patient EA
Paxalisib (formerly known as GDC-0084)
Disease/Category-Specific EA Policies/Criteria
https://www.kaziatherapeutics.com/researchpipeline/paxalisib
KC Pharma, LLC
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
KC Pharma DFMO EXPANDED ACCESS POLICY
KC Pharma (KCP) is committed to serving patients by providing safe, fair, and sustainable patient access to its medicines. KCP may be able to provide patients access to its investigational drug, DFMO (also called eflornithine or difluoromethylornithine), outside of a clinical trial if certain conditions are met and if permitted by local law and regulation. The use of an investigational medical product (i.e., one that has not been approved by the Food and Drug Administration [FDA]) outside of a clinical trial is known as Expanded Access (sometimes referred to as “compassionate use”). Generally, there are two types of Expanded Access—Group Expanded Access and Individual Patient Expanded Access. For more information on the different types of Expanded Access, visit the following FDA website: https://www.fda.gov/news-events/public-health-focus/expanded-access.
KCP collaborates with Beat Childhood Cancer Research Consortium (BCC) and Sponsor-Investigator, Dr. Giselle Sholler and makes investigational product DFMO 192 mg tablets available for pediatric oncology indications through multiple clinical trials and a Group Expanded Access Program (also called Intermediate Population Expanded Access). KCP does not currently provide Individual Patient Expanded Access.
Expanded Access may not always be available. Information about the Group Expanded Access Program, including a full list of eligibility criteria and site contact information can be found here: https://clinicaltrials.gov/ct2/show/NCT03581240. In addition to meeting the eligibility criteria outlined at the link above, the following criteria must be met for entry into the Group Expanded Access Program:
• KCP must have sufficient supply of DFMO to reasonably accommodate the anticipated duration of treatment.
• The patient’s licensed physician must be willing to collaborate with the enrolling center.
• The patient’s physician and enrolling physician determine there is no comparable or satisfactory therapy available to treat the patient’s disease or condition and they agree that the patient is clinically stable and able to receive this medication.
• The patient’s parent/guardian is willing to travel with the patient to BCC at study entry and every three months while on treatment.
Licensed physicians may contact KCP with general requests or questions regarding Expanded Access at DFMOExpandedAccess@usworldmeds.com. KCP will endeavor to acknowledge general requests and questions within 5 business days. In line with the 21st Century Cures Act, KCP cannot guarantee access to investigational product to all patients. KCP may revise this policy at any time; at such time, the revised policy will be made publicly available.
Krystal Biotech, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
We are privileged to collaborate with clinical investigators and patients who participate in our studies to develop new, safe and effective therapies. At the same time, we understand that there are patients who will not be eligible for our clinical trials and may not have options for effective alternative therapies. In these circumstances, Krystal will consider providing a requesting physician with pre-approval access to a specific Krystal investigational drug for the treatment of an individual patient outside of a clinical trial when certain conditions are met. These conditions include, but are not limited to, the following:
• The patient’s serious or life-threatening condition limits their ability to comply with certain clinical trial requirements, such as travel;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and medical history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials, regulatory review, or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.
We continually evaluate the safety and efficacy profile of each of our investigational drugs based on evolving clinical data. Each disease, patient, and investigation drug under development is unique, and as such, requests will be considered on a case-by-case basis.
Krystal is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Krystal may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Krystal, whose decisions are final.
Available Therapies via Single-Patient EA
Bercolagene Telserpavec (KB103) - Dystrophic Epidermolysis Bullosa
Disease/Category-Specific EA Policies/Criteria
https://www.krystalbio.com/wp-content/uploads/2019/07/Krystal-Expanded-Access-Policy.pdf
Kymab Limited
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Kymab is dedicated to developing novel human antibody-based therapeutics across a broad range of indications including immune mediated diseases.
Expanded access permits the use of an investigational therapy in patients with a serious or life-threatening illness when no satisfactory preventive or treatment options are available, and the patient is ineligible to participate in a clinical trial. Kymab understands and respects the need for appropriately managed expanded access program(s), and accordingly, this policy has been generated.
The following criteria are informed by regulatory guidelines for when expanded access may be made available to an investigational product on a case-by-case basis for individual patients:
• Ongoing or planned clinical studies are not available to the patient, including lack of access due to geographical location of potential clinical trial sites;
• Significant evidence exists that supports both the safety and the efficacy of the investigational drug for the indication;
• The potential benefits to the particular patient seeking access to the investigational drug outweigh the potential risks to the patient;
• An adequate supply of the investigational drug exists;
• All necessary regulatory/institutional approvals have been obtained to allow drug administration, and
• The request for expanded access has been made by a qualified health care provider with expertise appropriate for the administration of the drug and for monitoring and managing the patient.
KY1005 is currently in early clinical development. Kymab believes that the most appropriate way to ensure appropriate use of KY1005 at this stage in development is via approved clinical trials. The initial clinical trial(s) will permit the effective generation of safety and efficacy data for KY1005 which, when shared with the Regulatory Authorities will further guide the decision making regarding its safe and effective use in an expanded access program.
At this time, therefore, Kymab will not offer expanded access and will not currently accept expanded access requests for KY1005. Information regarding KY1005’s clinical trials will be made available at https://clinicaltrials.gov/.
If you have additional questions, please speak with your health care provider or contact KY1005ExpandedAccess@Kymab.com.
In accordance with the US 21st Century Cures Act, Kymab may revise this policy at any time.
Kyowa Kirin, Inc.
Single-Patient EA Policies/Criteria
Process for Requesting Expanded Access
Kyowa Kirin seeks to fulfill its management philosophy of striving to contribute to the health and wellbeing of people around the world by creating new value through the pursuit of advances in life sciences and technologies.
Physicians who are interested in treating a patient with a Kyowa Kirin unapproved drug and meet the criteria stated on our webpage must do the following:
1. Submit a request to Kyowa Kirin’s via email to the Access Mailbox: access@kyowakirin.com or via any Kyowa Kirin Medical Science Liaison with whom you have contact. The company will endeavor to acknowledge receipt of the request via email within five (5) business days of submission of the request.
2. Provide additional information as may be requested by Kyowa Kirin such as: relevant medical patient information, a persuasive scientific rationale for the theoretical benefit that the unapproved drug could provide to the patient, a statement no comparable or satisfactory alternative therapy options are available for the patient, including participation in ongoing relevant clinical trials.
Making a request does not guarantee the granting of access to an unapproved drug. Kyowa Kirin will review each request on a case-by-case basis. The decision to grant access is solely Kyowa Kirin’s decision. Kyowa Kirin reserves the right to terminate the supply of drug at any time.
Contact for Further Information and Link to ClinicalTrials.Gov
Persons with questions about Kyowa Kirin’s policy for expanded access or about other issues related to expanded access to unapproved drugs may contact the company at the Access Mailbox: access@kyowakirin.com.
Further information about Kyowa Kirin’s clinical trials in the U.S. is available on the NIH’s ClinicalTrials.gov website.
Laminar Pharma Inc
Phone Number & Email
Single-Patient EA Policies/Criteria
LaNova Medicines Limited
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
Process for Requesting Access
- If you are not eligible for a clinical trial or no appropriate clinical trial exists, and your qualified treating physician believes that an investigational medicine available may be the best and the only treatment option for you, your physician should contact us on your behalf by emailing LM-108EA@lanovamed.com. Our physician will acknowledge the requests within 5 business days of receipt.
- The company may request additional information, including patient history, in order to fully evaluate the request.
Available Therapies via Single-Patient EA
Expanded Access
- We understand the interest of patients in accessing LM-108 and other novel investigational medicines outside of clinical trials and prior to regulatory approval for potential life-threatening diseases, however we do not currently offer Expanded Access Programs for our investigational medicines as all of them are still in early phase clinical stage.
- In very rare and specific circumstances, patients with serious or life-threatening disease may still need access to our investigational medicines. Please consult with your physician, the requests will be reviewed with our physician case by case. Several important medical and logistic criteria need to be met including:
- The disease is serious or life-threatening;
- All other available alternative therapies have been exhausted, but the patient may still benefit from further treatment with an investigational medicines;
- A clinical trial is not available, either because patients are ineligible or because they have no access to the trial;
- The efficacy and safety data available at the time are of sufficient strength to determine whether the benefit to the patient would likely outweigh the potential risks; and
- Access is compliant with all applicable laws and regulations.
- There is an adequate supply of the investigational medicine in local.
- There is an ongoing clinical trial in local which can support the investigational medicines distribution.
- Regulatory requirements have been completed based on local IRB requirements, such as compassionate use.
LaNova Medicines Limited
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
Process for Requesting Access
- If you are not eligible for a clinical trial or no appropriate clinical trial exists, and your qualified treating physician believes that an investigational medicine available may be the best and the only treatment option for you, your physician should contact us on your behalf by emailing LM-108EA@lanovamed.com. Our physician will acknowledge the requests within 5 business days of receipt.
- The company may request additional information, including patient history, in order to fully evaluate the request.
Available Therapies via Single-Patient EA
Expanded Access
- We understand the interest of patients in accessing LM-108 and other novel investigational medicines outside of clinical trials and prior to regulatory approval for potential life-threatening diseases, however we do not currently offer Expanded Access Programs for our investigational medicines as all of them are still in early phase clinical stage.
- In very rare and specific circumstances, patients with serious or life-threatening disease may still need access to our investigational medicines. Please consult with your physician, the requests will be reviewed with our physician case by case. Several important medical and logistic criteria need to be met including:
- The disease is serious or life-threatening;
- All other available alternative therapies have been exhausted, but the patient may still benefit from further treatment with an investigational medicines;
- A clinical trial is not available, either because patients are ineligible or because they have no access to the trial;
- The efficacy and safety data available at the time are of sufficient strength to determine whether the benefit to the patient would likely outweigh the potential risks; and
- Access is compliant with all applicable laws and regulations.
- There is an adequate supply of the investigational medicine in local.
- There is an ongoing clinical trial in local which can support the investigational medicines distribution.
- Regulatory requirements have been completed based on local IRB requirements, such as compassionate use.
Leap Therapeutics, Inc.
Expected Application Timeframe
5 business daysDisease/Category-Specific EA Policies/Criteria
Life Molecular Imaging Ltd (LMI)
Single-Patient EA Policies/Criteria
LMI is not offering expanded access to PI-2620 or other products at this time.
Linnaeus Therapeutics
Expected Application Timeframe
7 daysSingle-Patient EA Policies/Criteria
Expanded Access Program Policy for LNS8801
LNS8801 is currently being developed for the treatment of advanced cancer and has fast track designation for the treatment of patients with metastatic or unresectable melanoma who have progressed on or after anti–programmed cell death receptor or ligand (anti–PD-1/L1) therapy. Linnaeus Therapeutics (Linnaeus) understands that advanced cancer patients with limited therapeutic options may desire to try promising experimental therapies such as LNS8801. The best way for patients to get access to investigational medicines is by taking part in clinical trials. Please search ClinicalTrials.gov for publicly available information related to Linnaeus’s ongoing clinical trials.
It is recognized that not all patients are eligible to enroll in clinical trials. Linnaeus may consider requests for expanded access (sometimes called compassionate use) at this time due to limited drug supply.
Linnaeus hopes that in the near future it will be able to regularly consider requests so that patients in the United States with an immediately life-threatening condition or serious disease or condition who have exhausted other appropriate treatment options may, under the conditions described below and in accordance with applicable local law, have appropriate access to its investigational medicines in development before they are commercially available.
For more information regarding Linnaeus’ current and future expanded access policies please contact, medinfo@linnaeustx.com. Linnaeus anticipated being able to response to inquiries within one week.
Available Therapies via Single-Patient EA
LNS8801 - advanced cancers
Lion TCR
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
SCOPE
This policy applies to provision of access to Lion TCR’s investigational product(s) that is not approved for any purpose in the country from which the request is intended to be used. This also includes the time period between regulatory approval of an investigational product and its commercial availability in a country.
POLICY STATEMENTS
Any use of Lion TCR’s investigational product(s) outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including our policies and procedures.
In general, where permitted by local regulation, the investigational product supplied via Expanded Access may no longer be provided by Lion TCR when it becomes available via the local healthcare system.
Lion TCR may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the country.
A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Lion TCR's investigational products by contacting the Company at clinicaltrials@liontcr.com . We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 5 business days after receipt.
Lion TCR is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Lion TCR may require more detailed information in order to fully evaluate a request. Requests will be considered on a case-by-case basis.
Lion TCR will consider providing a requesting physician with pre-approval access to a specific investigational drug outside of a clinical trial, when the following conditions are met:
A. Patient Eligibility Criteria
To be eligible for access to an investigational product, patients must meet the following criteria:
- Suffer from a serious or immediately life-threatening disease or condition.
- Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
- Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
- The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
- Any other pertinent medical criteria for access to the investigational product, as established by the Lion TCR clinically or medically responsible individual.
B. Investigational Product Criteria
In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
- The product is under investigation in one or more clinical studies.
- There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
- The provision of the investigational product will not interfere with or compromise the clinical development of the product.
C. Treating Physician Criteria and Responsibilities
The physician(s) attending to the patient(s) who is/are receiving an investigational product through compassionate use access is (are) properly licensed and fully qualified to administer the product. The physician must agree in writing to comply with:
- Any applicable country-specific legal and regulatory requirements related to providing an investigational product under Expanded Access.
- Any requirements from Lion TCR in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property.
Available Therapies via Single-Patient EA
LioCyx-M for hepatocellular carcinoma
Disease/Category-Specific EA Policies/Criteria
https://drive.google.com/file/d/1d9bjsBW4w7WpsBFcXEZT8fwd8OvB8GAx/view
Lisata Therapeutics, Inc.
Expected Application Timeframe
1-3 business daysAvailable Therapies via Single-Patient EA
Currently, Lisata does not make its unapproved drugs available on an expanded access basis. Lisata does, however, encourage interested individuals to contact the Company regarding potential participation in its clinical trials.
Lumicell
Single-Patient EA Policies/Criteria
At Lumicell, our focus is on the development of pegulicianine. The goal of our current clinical study program is to enroll patients and obtain clinical data on pegulicianine that is required by the US Food and Drug Administration (FDA) and other regulatory authorities for review and approval of marketing applications for this investigational agent. We believe that focusing on clinical studies is the most appropriate way to achieve this goal; therefore, we do not currently offer access to pegulicianine outside of clinical studies and, at this time, we have no Expanded Access program (sometimes referred to as “compassionate use” or “pre-approval access”).
For information on participating in and status of pegulicianine clinical trials, please see: https://clinicaltrials.gov/ct2/home
If you have additional questions regarding this policy, please speak with your health care provider or contact: info@lumicell.com
Lundbeck
Expected Application Timeframe
10 business daysMacroGenics, Inc.
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
MacroGenics designs its clinical studies for development of new potential therapies to determine the safety and efficacy for medical use consistent with approval requirements of the US FDA and similar regulatory authorities in other countries. Treating physicians should consider approved therapies for a patient’s disease as well as on-going clinical studies before seeking expanded access use with an investigational agent.
However, MacroGenics may, on a case-by-case basis, consider requests by treating physicians to file an investigational new drug application for expanded access to MacroGenics investigational products in Phase 2 or Phase 3 of development and for MacroGenics to supply such investigational product. There is no guarantee of expanded access to an investigational product.
Available Therapies via Single-Patient EA
At this time, MacroGenics is unable to make investigational products available outside a clinical trial.
Marinus Pharmaceuticals
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
Patients must satisfy the following criteria to be enrolled in the expanded access protocol:
Main Inclusion Criteria:
1. Molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required.
2. Male or female patients aged ≥ 2 years.
3. Informed Consent signed by the patient, patient’s parent(s) or LAR indicating that they understand the purpose of and procedures required for expanded access and are willing to participate in the expanded access protocol.
4. Age-appropriate assent, if indicated.
5. In the opinion of the Investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses.
6. Able and willing to take investigational product (oral suspension) with food 3 times daily (TID). Ganaxolone must be administered with food.
Exclusion Criteria:
1. Pregnant or breastfeeding.
2. Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging).
3. Known allergic reaction or sensitivity to GNX or excipients.
4. Previously or actively participating in a clinical trial of GNX.
5. Participating in any study involving administration of an investigational agent, excluding devices.
MEDRx USA., Inc.
Single-Patient EA Policies/Criteria
MEDRx USA, Inc. (MEDRx) is a clinical-stage pharmaceutical company that focuses on research and development of proprietary investigational products.
Investigational drug products generally can only be provided to subjects enrolled in clinical studies as such products are not approved by the U.S. Food and Drug Administration (“FDA”) as safe and effective for their intended use. While investigational product manufacturers may provide investigational products to certain patients under FDA’s Expanded Access Program (sometimes called “compassionate use”), if the patients meet certain criteria required by FDA law and regulation, at this time, MEDRx will not offer expanded access to investigational products outside of enrollment in clinical trials and is currently not accepting expanded access requests. If you have questions or need more information about our programs, please feel free to contact MEDRx at info@medrxusa.com . MEDRx endeavors to respond within five business days.
In line with the 21st Century Cures Act, MEDRx may revise this policy at any time.
Meissa Vaccines Inc
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
Meissa Vaccines, Inc. (https://www.meissavaccines.com) is developing investigational vaccines for the prevention of respiratory diseases. Since the vaccines are designed for prophylactic not treatment use, Meissa has chosen not to offer Expanded Access to these vaccines and will not accept Expanded Access requests. Meissa’s investigational vaccines will only be provided through ongoing clinical trials. To learn more about available clinical trials, please visit https://www.meissavaccines.com, or visit https://clinicaltrials.gov and search using the company name.
Meissa Vaccines, Inc. Expanded Access Policy, rev. August 2021.
Melinta Therapeutics, Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Consistent with Melinta Therapeutics’s mission to discover, develop, and commercialize a continual stream of novel antibiotics, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals and promptly make our medicines available to patients. At Melinta Therapeutics, we believe that wherever possible, the use of an investigational medicine for a patient as part of a clinical trial is preferable; however, when patients with an unmet medical need are unable to access our investigational products through a clinical trial and all other options have been exhausted, we may be able to provide access through our compassionate use or expanded access program.
The evaluation criteria to determine if Melinta Therapeutics can provide expanded access to a specific Melinta Therapeutics investigational agent outside of a clinical trial include all of the following:
• The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Access is compliant with local rules and laws;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.
All requests must be submitted by the patient’s treating physician and Melinta Therapeutics may require more detailed information in order to fully evaluate a request. The treating physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Treating physicians seeking expanded access, meeting the above-mentioned evaluation criteria, should submit their requests to medinfo@melinta.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt. Melinta Therapeutics is committed to carefully evaluating requests on a case-by-case basis and in a fair and equitable manner; however, Melinta Therapeutics cannot guarantee access to any specific investigational drug by any individual patient.
If you are a patient with questions concerning access to our investigational products, either through a clinical trial or through expanded access, please consult with your treating physician.
Mendus
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Currently, Immunicum does not have any investigational cell therapy products
available for Expanded Access
Available Therapies via Single-Patient EA
Immunicum is currently unable to offer an Expanded Access
Merck & Co.
Expected Application Timeframe
N/AMerus NV
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Criteria for Evaluation
Merus considers many factors when evaluating a request for early access of an investigational medicine, such as (but not limited to) the strength of the currently available data, including clinical data together with patient condition and the benefit-risk profile, the impact of providing access to an investigational medicine on its clinical development program, including the ability to complete patient enrollment and other necessary aspects of the clinical trials, the phase of development, and probability and timing of regulatory approval.
A patient may be considered for early access to an investigational medicine based upon evaluation of the following minimum requirements (and other requirements may apply based on the circumstances):
*There must be adequate data supporting use of the investigational medication in the treatment of the patient’s disease.
*There are no adequate standard or investigational therapies available.
*Sufficient safety and efficacy data must exist for use of the medicine at the time of request.
*Sufficient information must be made available to make a benefit-risk analysis consistent with the establishment of an early access program.
*Sufficient clinical data is available to identify an appropriate patient-specific dose.
*A patient’s treating physician and Merus’ clinical team both believe there is the potential for the specific patient under consideration to reasonably expect benefit from the treatment.
*Adequate supply exists to support both the ongoing clinical trials and approved early access, until and if a product becomes commercially available.
*The patient is not eligible for or cannot participate in a Merus-sponsored study for the therapy being requested.
*Early access will not adversely impact the clinical development program or the regulatory approval process.
*The request must be made, unsolicited, by the patient’s treating physician. The physician must be appropriately licensed.
*The treating physician must also agree to comply with the safety and monitoring requirements.
*Where appropriate, demonstration of efficacy and safety may be required for continued treatment.
*Early access is permitted or approved in the country, region, and/or state where the investigational medicine will be administered.
*Any condition, in addition to those listed above, that in the opinion of Merus may preclude appropriate use of the investigational medicine, will be considered.
Given the unique nature of individual requests for early access to investigational medications, there is no guarantee a request will be fulfilled.
Merus may revise, suspend or terminate the early access program at any time.
All early access requires approval by the Chief Medical Officer and Merus management and decisions will be made based on scientific and other evidence available to Merus at the time of the request.
Available Therapies via Single-Patient EA
MCLA-128 is available for patients with solid tumors that harbor an NRG1 fusion
Disease/Category-Specific EA Policies/Criteria
Metacrine
Single-Patient EA Policies/Criteria
At this time, Metacrine believes access to its products should be limited to clinical trials and does not offer an expanded access program or accept expanded access requests.
MetVital, Inc.
Expected Application Timeframe
72 hours after receipt of a written requestSingle-Patient EA Policies/Criteria
Met Vital, Inc. will consider providing pre-approval access to our investigational drug, outside of the clinical trial setting, when certain conditions are met. These conditions are as follows:
• The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy.
• The patient is not eligible for, or cannot access, any ongoing clinical trials.
• The potential benefit of the investigational medicine to the patient, outweighs the potential risk. This should be evaluated by your physician and discussed in detail with the patient.
• There is an adequate supply of the investigational drug, meaning that MetVital, Inc has surplus investigational drug over and above what is required for the ongoing clinical trial.
• Providing the investigational drug will not interfere with the clinical trial that could support a marketing approval.
Requests for our investigational drug must come from a patient’s treating physician. The requesting physician must be willing to work on designing a treatment protocol and obtain the appropriate regulatory and ethics committee approvals from their Institutional Review Board (IRB). MetVital, Inc will not be responsible for the physician gaining IRB approval. The physician must also comply with various regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting.
Available Therapies via Single-Patient EA
Anhydrous enol-oxaloacetate for newly diagnosed Glioblastoma multiforme
Disease/Category-Specific EA Policies/Criteria
Millendo Therapeutics (US), Inc.
Expected Application Timeframe
15 daysAvailable Therapies via Single-Patient EA
Currently, none.
Minoryx Therapeutics
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Minoryx is a clinical stage biotech company committed to the development of safe and effective novel therapies for severe and life-threatening orphan CNS diseases with high unmet medical needs. We are a science-driven company committed to putting patients first. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Clinical trials and the subsequent timely generation of safety and efficacy data are the most effective way of ensuring review and decision making by Regulatory Authorities. This ultimately has resulted in access to new, safe and effective approved medicines for patients.
Expanded access refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.
Disease/Category-Specific EA Policies/Criteria
FDA Orphan indication was granted for the company’s lead program, leriglitazone (MIN-102), a novel, selective peroxisome proliferator-activated receptor gamma (PPARγ) agonist, for the treatment of X-ALD. On 4 April 2018, Minoryx opened the IND for leriglitazone (company code MIN-102) with a double-blind, randomized, placebo-controlled study to assess the effect of leriglitazone on the clinical progression in male patients with Adrenomyeloneuropathy (AMN). Recently, the FDA granted Fast Track Status to leriglitazone for development in patients with X-ALD.
An Early Access program for leriglitazone may be opened if Minoryx determines that all the following criteria are met:
• The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
• An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
• Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
• There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient
At this time, Minoryx believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. Minoryx continues to assess the eligibility requirements and criteria for Early Access to the investigational drug leriglitazone and will re-evaluate this policy from time to time.
If you have additional questions, please speak with your physician or contact clinical@minoryx.com. We anticipate acknowledging receipt of requests sent to this email within five business days.
Minovia Therapeutics
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Minovia will make every attempt to respond to EAP request as soon as possible. An acknowledgement of receipt of request will be made within one week. Responses as to relevance of patient for EAP treatment may vary, based on the nature of the request and details of rationale for patient not being eligible for the currently open clinical trial.
Written requests from the patient’s healthcare provider should be submitted by email. All requests will remain confidential.
Disease/Category-Specific EA Policies/Criteria
Patients will be considered for EAP if they are not eligible for the currently open clinical trial of MNV-BM-BLD, carry a mitochondrial DNA deletion, and the mother is not a carrier of this deletion. Patient ineligibility for the open clinical trial may be due to their not meeting all inclusion/exclusion criteria (eg patient is below minimum age) or having a mitochondrial deletion syndrome other than Pearson Syndrome.
Healthcare providers interested in information about the MNV-BM-BLD product for their patients can contact Minovia to request expanded access at expandedaccess@minovia.com
Mirati Therapeutics
Expected Application Timeframe
1 business daySingle-Patient EA Policies/Criteria
Single-Patient Expanded Access Policies and Criteria - A licensed physician who believes their patient may benefit from access to a Mirati investigational medicine outside of a clinical trial should contact Early Access Care, by email Mirati.ExpandedAccess@earlyaccesscare.com or by contacting the Mirati Expanded Access Call Center 1-475-522-2200.
Available Therapies via Single-Patient EA
Expanded Access of Adagrasib (MRTX849) in Patients With Advanced Solid Tumors Who Have a KRAS G12C Mutation
Conditions: Advanced solid tumors harboring a KRAS G12C mutation
This is a multi-center, expanded access protocol to provide access to the investigational product, MRTX849, to adult patients with advanced solid tumors who have inadequate treatment options with available and approved therapies and who are otherwise ineligible for participating in clinical studies with MRTX829.
Disease/Category-Specific EA Policies/Criteria
https://www.earlyaccesscare.com/companies/mirati-therapeutics/adagrasib
ModernaTX, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Moderna is working to deliver on the promise of mRNA science to create a new class of medicines for patients. As we advance our pipeline, patients, caregivers and healthcare providers may seek access to our investigational medicines outside of clinical trials and prior to regulatory approval. We understand the need for new therapies and the urgency behind requests for pre-approval access*.
While we continue evaluating the safety and effectiveness of this potential new class of medicines in clinical trials, we are not providing access to our investigational mRNA therapies or vaccines outside of these studies. We believe that participation in a clinical trial is the most appropriate way for patients to access any of our investigational treatments. We also believe that participation in clinical trials is the most efficient path to providing access to the greatest number of patients in need of a new therapy. Should our policy change over time, we will post updates on this site.
You can find links to information about our ongoing clinical trials on our website or go directly to www.clinicaltrials.gov and enter “Moderna” into the “other terms” search box. Please speak with your physician if you have any additional questions about our clinical trials or this policy. Our team plans to acknowledge receipt of requests sent to this email address from patients, families and healthcare providers within 5 business days.
*Includes requests for access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, as well as ‘Right to Try’ legislation (Public law no. 115-176).
Molecular Partners AG
Single-Patient EA Policies/Criteria
Molecular Partners AG is a clinical-stage biotech company developing DARPin® therapeutics, a new class of custom-built protein drugs designed to address challenges current modalities cannot. Molecular Partners develops DARPin® therapeutics for infectious disease, oncology including hematological malignancies, and ophthalmology. Ensovibep is currently being investigated in clinical trials for patients suffering from COVID-19 infection.
We at Molecular Partners, have a patient-focused approach in our development of medicines and we need to conduct clinical trials in order to gain regulatory approval, making ensovibep available to patients as quickly as possible. Molecular Partners understand the interest of patients in accessing ensovibep outside of clinical trials and prior to regulatory approval for the treatment of COVID-19. However, with alternatives which have achieved market approval or Emergency Use Authorization by the FDA these are more appropriate alternatives at current and we do not currently offer Expanded Access programs for ensovibep.
We recognize the need for Expanded Access programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.
Information about our clinical trials, including eligibility criteria and locations, is available at www.clinicaltrials.gov.
Momenta
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Momenta is committed to developing safe and effective medicines to treat rare immune-mediated diseases with high unmet medical needs. Our goal is to ensure access to our investigational drug, at the appropriate time and in a clinically appropriate manner for patients.
Momenta’s Early/Expanded Access Policy (EAP) refers to the use of an investigational drug outside of a clinical trial. This is initiated when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient, which is different from a clinical trial where more comprehensive safety and efficacy data are collected. At Momenta, we recognize and understand the need for an early/expanded access policy.
A number of factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies' guidelines should be taken into account when considering this kind of access.
Momenta’s Key Eligibility Requirements
To be eligible to access Momenta's investigational drugs under the EAP, participants must meet certain requirements, including, but not limited to:
- The illness must be serious or life-threating, with no other satisfactory treatment options (such as approved products or enrolling clinical trials)
- There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on all available safety and efficacy information
- Providing the investigational drug will not interfere with clinical trials that could support the investigational drug's development or marketing approval for the treatment indication
At this time, Momenta believes that participation in one of our clinical trials is the most appropriate way to access our investigational drugs. We do not currently provide our investigational drugs for use through expanded access.
MorphoSys
Expected Application Timeframe
2-3 business daysSingle-Patient EA Policies/Criteria
If you are a patient and interested in obtaining access to an investigational medicinal product, you should discuss available options with your treating physician. If a MorphoSys clinical trial may be a good treatment option for you, we recommend that your treating physician contacts us on your behalf via the contact information provided in ClinicalTrials.gov (search for Tafasitamab).
If you are not eligible for a clinical trial that is currently active, or if no appropriate clinical trials exist, please contact your treating physician to determine if an EAP may be the best or only treatment option for you. Your physician will find details on the EAP at ClinicalTrials.gov with the identifier NCT04300803.
Treating physicians should use the following contact (tafasitamab@clinigengroup.com) to receive the necessary request form, eligibility criteria and instructions for the EAP process. Receipt of requests will be acknowledged within two business days of receipt, approval or denial will usually be finalized within 2-3 business days. Before treatment can be started, the treating physician has to obtain IRB review.
MorphoSys is committed to a fair and impartial evaluation of each request for access to our EAP. Therefore, all decisions are based solely on clinical evidence and guided by the principles outlined below and by eligibility criteria for the EAP. In addition, MorphoSys intends to work in cooperation with local health authorities for the provision of an EAP.
Available Therapies via Single-Patient EA
Tafasitamab
Tafasitamab is used in combination with lenalidomide for the treatment of adult patients with relapse or refractory diffuse large B cell lymphoma (R/R DLBCL)
Myeloid Therapeutics
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Myeloid has one product, MT-101, in clinical trials. The trial is a Phase 1 multi-dose ascending trial for safety and tolerability. There is no expanded access to MT-101 until more therapeutic data is obtained.
Disease/Category-Specific EA Policies/Criteria
NanoCarrier Co., Ltd.
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
A. Patient Eligibility Criteria
To be eligible for access to an investigational product, patients must meet the following criteria:
• Suffer from a serious or immediately life-threatening disease or condition.
• Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• There is adequate information to support appropriate dosing for a special patient population such as pediatric, elderly, renal or hepatic disease, etc.
• Any other pertinent medical criteria for access to the investigational product, as established by the NanoCarrier clinically or medically responsible individual.
B. Investigational Product Criteria
In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
• The product is under investigation in one or more clinical studies.
• There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
• The provision of the investigational product will not interfere with or compromise the clinical development of the product.
Available Therapies via Single-Patient EA
NC-6300 (nanoparticle epirubicin) for the treatment of patients with advanced or metastatic angiosarcoma not amenable to curative treatment with radiation or surgery
Disease/Category-Specific EA Policies/Criteria
https://www.nanocarrier.co.jp/en/pipeline/expanded_access_policy/
NeoMatrix Therapeutics, Inc.
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
NeoMatrix Therapeutics (the Company) is the developer of a novel investigational drug product, cP12. The investigational drug product is being evaluated for the treatment of serious burn injuries and is still in the early stages of clinical development.
At the current stage of clinical development, NeoMatrix Therapeutics has chosen not to offer Expanded Access (EA) and will not accept EA requests for the investigational drug product. The justification of the restriction on EA is as follows:
• There is insufficient safety and efficacy data to determine the risk-benefit profile for the treatment of serious burn injuries.
• The Company’s ability to conduct clinical trials is limited. Offering EA under any setting at this time will impact the Company’s ability to execute its trials.
• The organization does not have the personnel and other resources to offer EA on a fair and equitable basis.
• The Company’s investigational drug product cP12 requires special handling and shipping.
As the company is not currently accepting EA requests, the specified policy information is not provided at this time. The status of EA restriction will be re-evaluated if early data from future clinical trials suggest that cP12 investigational treatment can offer benefits for patients facing serious or life-threatening conditions.
NeoMatrix Therapeutics, Inc. Expanded Access Policy, Rev: May 17, 2019
Neuraptive Therapeutics, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
At Neuraptive, we are committed to developing first-in-class treatments for patients with peripheral nerve injuries (PNI) and physicians who treat them.
NTX-001 has been designed and is being developed as an adjunctive treatment for patients with nerve injuries and may restore and improve the recovery of neurons following nerve repair surgery. We will be conducting clinical studies to demonstrate the safety and efficacy of NTX-001, and to obtain regulatory approval and ultimately make NTX-001 available.
Before regulatory approval by the U.S. Food and Drug Administration (FDA) and foreign regulatory authorities, patients gain access to investigational treatments, such as NTX-001, by participating in clinical trials and by ‘Expanded access,’ also referred to as compassionate use, ‘Early Access,’ and ‘Emergency Use’. Patients who seek access to investigational medicines outside of an established clinical study and before health authority marketing authorization may wish to do so because standard treatments have failed, because they cannot tolerate already approved medicines, because they are unable to participate in a clinical study or because there are no comparable or satisfactory therapy options available outside of clinical trials. The primary purpose of expanded access is to use the investigational drug for patient treatment purposes rather than to gather data on safety, tolerability, and effectiveness.
Currently, Neuraptive does not offer an expanded access program and does not accept expanded access requests outside of clinical trials. We believe that access to NTX-001 should be limited to clinical trials until its safety, tolerability, and effectiveness for a particular indication has been determined and confirmed by regulatory authorities. Patient access to NTX-001 outside of a clinical trial may interfere with the conduct of our ongoing and future clinical trials, and may also disrupt the progress of our development program, which would, in turn, delay access to many patients in need.
Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials is the only safe and appropriate way to access NTX-001.
If you have questions about Neuraptive’s expanded access policy, please contact Neuraptive at info@Neuraptive.com. Please expect a response within five business days.
In line with the 21ST Century Cures Act, Neuraptive may revise this policy at any time.
NH TherAguix
Available Therapies via Single-Patient EA
At NH TherAguix, our mission is to develop novel nanomedicine solutions applicable to precision radiotherapy in oncology. Our drug candidate AGuIX® is a nanodrug designed to meet the growing medical needs in brain cancer by improving the efficacy and precision of radiotherapy directly within tumors.
As defined by the US Food and Drug Administration, expanded access, sometimes called “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Our goal is to provide access to our investigational drug at the appropriate time and in a manner that is most beneficial to the relevant patient population. We understand that patients, caregivers, or health professionals may wish to have access to our drug candidate before receive regulatory approval, outside the context of a clinical trial. At the present time, we are not making our product available for such pre-approval access. We believe enrollment in our ongoing clinical trial is the safest and most effective way of achieving this goal. We currently have ongoing Phase 1b/2 and Phase 2 clinical trials, in which the investigational drug is added to the standard of care that patients are already be receiving. You can find information about ongoing clinical trials of our drug candidate by visiting www.clinicaltrials.gov and entering “AGuIX” in the “other terms” search box.
NH TherAguix may revisit this policy as we gain additional scientific knowledge about our drug candidate through clinical trial data. Any such changes will be posted to this site.
Novartis Pharmaceuticals
Expected Application Timeframe
ImmediatelySingle-Patient EA Policies/Criteria
Novartis considers granting managed access to investigational or pre-approval products via single patient access provided the Managed Access criteria are fulfilled.
Disease/Category-Specific EA Policies/Criteria
Product |
Indication |
Capmatinib |
MET dysregulated Non-Small Cell Lung Cancer (NSCLC) |
Ceritinib |
Non-small cell lung cancer: ALK positive tumors, previously pre-treated with ALK inhibitors or naive |
Clofazimine |
Mycobacterial infection |
Iptacopan |
C3 Glomerulopathy (C3G) |
Tisagenlecleucel |
Acute lymphoblastic leukaemia; Diffuse large B-cell lymphoma refractory |
Novavax, Inc
Phone Number & Email
Expected Application Timeframe
5 Business DaysAvailable Therapies via Single-Patient EA
Novavax promotes improved health by discovering, developing, and commercializing innovative vaccines to protect against serious infectious diseases. We offer a differentiated vaccine platform that combines a recombinant protein approach, innovative nanoparticle technology and Novavax's patented Matrix-M adjuvant to enhance the immune response.
Our vaccines are designed to be used prophylactically, meaning that they are designed and intended to prevent disease in healthy individuals, not for the treatment of existing disease. Like all vaccines, our products must be carefully evaluated in clinical trials to ensure that they can be used safely and effectively.
We understand that patients, caregivers, or healthcare professionals may wish to have access to our vaccine candidates before they receive regulatory approval, outside the context of a clinical trial. At the present time, we are not making our products available for such pre-approval access. This includes requests for pre-approval access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, the Right to Try Act of 2017 (Pub. L. No. 115-176), or any state laws. We believe that participation in clinical trials, with their established eligibility criteria and ethical safeguards, is the most appropriate and equitable approach to making our investigational vaccines available before they are approved.
Novavax may revisit this policy as we gain additional scientific knowledge about our vaccine products through clinical trial data and real-world use after vaccine licensure or authorization, or if we develop therapeutic products in the future. Any such changes will be posted to this site.
You can find information about ongoing clinical trials of our vaccines by visiting www.clinicaltrials.gov and entering “Novavax” in the “other terms” search box. Any inquiries regarding this policy should be directed to https://www.novavax.com/contact-us. Novavax expects to acknowledge any such inquiries from patients, caregivers, or healthcare professionals within five business days.
Novo Nordisk
Expected Application Timeframe
5 business daysNS Pharma, Inc.
Expected Application Timeframe
Seven (7) business daysSingle-Patient EA Policies/Criteria
Single-Patient Expanded Access Policies and Criteria - If a treating physician believes that expanded access may be the only option for a patient, the physician should contact NS Pharma to make a formal request on behalf of a patient. Physicians may contact Early Access Care by emailing nspharma.expandedaccess@earlyaccesscare.com. We may request additional information to assist with patient eligibility for an investigational medication.
Available Therapies via Single-Patient EA
Viltolarsen Expanded Access for Duchenne Muscular Dystrophy - Confirmed DMD mutation in the dystrophin gene that is amenable to skipping of exon 53
Disease/Category-Specific EA Policies/Criteria
https://www.earlyaccesscare.com/companies/ns+pharma+inc./viltolarsen
Nuvation Bio, Inc.
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
At this time, Nuvation Bio products are considered investigational, which means that they have not been approved as safe and effective by regulatory health authorities, such as the United States Food and Drug Administration (FDA). Currently, participation in clinical trials is the only way for patients to gain access to Nuvation Bio’s investigational drugs. As more clinical data on the safety and efficacy of these investigational drugs become available, Nuvation Bio will review and update its policy on Expanded Access. For information on Nuvation Bio’s clinical trials, please email ClinicalTrials@nuvationbio.com.
Nuvectis Pharma, Inc.
Single-Patient EA Policies/Criteria
On our website (www.nuvectis.com) physicians and patients may explore information about our investigational drug candidate, NXP800.
Given the early stage of the NXP800 development program, we believe that participation in our clinical trial(s) is the most appropriate way to access NXP800, and therefore we are currently not making NXP800 available through an expanded access program.
In line with the 21st Century Cures Act, Nuvectis may revise this policy at any time.
Nuvig Therapeutics, Inc
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
At this time, given the early stage of development, Nuvig Therapeutics is not offering Expanded access for NVG-2089
Oblato, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Oblato Inc. will consider individual single patient access to OKN-007 in the United States for patients with Diffuse Intrinsic Pontine Glioma (DIPG), who cannot be treated by currently available drugs or clinical trials.
The primary option for patients to get access to investigational drugs is to participate in a clinical trial. Patients and physicians can learn more by reviewing information on current or planned clinical trials that is maintained by the U.S. National Institutes of Health.
We recognize, though, that there are extraordinary circumstances where access to these investigational drugs outside of a clinical trial is appropriate. Under our Access to Investigational Drugs Policy, a patient’s physician can request consideration for access if five basic criteria below are met:
- Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- Patient enrollment in a clinical trial is not possible.
- Potential patient benefit justifies the potential risks of treatment.
- Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
Available Therapies via Single-Patient EA
OKN-007 for patients with Diffuse Intrinsic Pontine Glioma (DIPG)
On Target Laboratories, Inc.
Expected Application Timeframe
3-5 business daysSingle-Patient EA Policies/Criteria
Pafolacianine sodium injection (OTL38) can be accessed through On Target’s Expanded Access program by contacting On Target Laboratories directly. The patient would be required to travel to an approved clinical site, where the investigator has completed the necessary training to administer the drug and has access to the required Near-Infrared Camera device. This would include any clinical investigator who have been approved to participate and enrolled subjects in either the Phase 2 or Phase 3 Lung trials with the drug. The patient may qualify to be enrolled in an open study, if available, and On Target would not include this subject in the randomization plan and their data would not be included in the efficacy analysis of the open study. In the event this is not possible, an Expanded Access Submission would be required to be submitted under the open IND 118215 as a Protocol Amendment. On Target Laboratories would work with the selected clinical investigator to generate the submission, which would include the required information defined in CFR 312.305, including the rational for the intended use of the drug, criteria for the patient selection, and the benefit risk of utilizing the investigational drug and device. The patient selection criteria would match the inclusion criteria defined in the Lung Cancer Phase 3 trial.
Available Therapies via Single-Patient EA
Pafolacianine sodium injection (OTL38) for use as an adjunct imaging agent in the detection of lung cancer during surgery.
Oncoceutics, Inc.
Expected Application Timeframe
48 hoursSingle-Patient EA Policies/Criteria
Oncoceutics will consider single - patient expanded access for patients who meet the criteria for the company's existing expanded access program, as outlined on clinicaltrials.gov
Available Therapies via Single-Patient EA
ONC201 - H3 K27M mutant gliomas
Oncoinvent ASA
Single-Patient EA Policies/Criteria
Expanded access is currently not available.
OncoNano Medicine Inc.
Single-Patient EA Policies/Criteria
At OncoNano Medicine, we are committed to developing products that bring new, innovative therapies, like pegsitacianine (ONM-100), to patients with serious or life-threatening illnesses or conditions. OncoNano is developing pegsitacianine to improve the ability of surgeons to identify cancerous tissue in the peritoneum during cytoreductive surgery.
At this time, OncoNano does not offer an expanded access program and is not accepting expanded access requests for investigational products, such as pegsitacianine.
OncoNano’s current focus and priority is to complete the product development program for its investigational product(s), such as pegsitacianine, in order to obtain the required safety and efficacy data needed for regulatory approval. We believe that focusing our resources on our clinical trial program is the best path forward to bring our investigational product(s) to patients as quickly and safely as possible.
As we continue to develop investigational product(s), we will review our expanded access policy for investigational product(s) and may make updates to this policy.
Patients can gain access to our investigational product(s), such as pegsitacianine, by participating in a clinical trial. If you or someone you know would like to learn more about OncoNano Medicine’s clinical trials, we encourage you to view our trials at www.clinicaltrials.gov.
If you have additional questions about OncoNano’s expanded access policy, please email us at info@onconanomed.com.
Oncopeptides, AB
Expected Application Timeframe
24 hoursOncoverity
Expected Application Timeframe
2 business daysSingle-Patient EA Policies/Criteria
To Whom It Concerns,
OncoVerity, Inc.’s (“OncoVerity” or “we”) mission is to reimagine a world where Cancer never wins. We
understand that success can only be achieved by collaborating closely with the families, patient groups,
clinicians, and regulators who share our mission of initially serving patients with AML.
Our focus is to advance Cusatuzumab toward regulatory approval and make it available to all who can
benefit from this therapy. Clinical research in the form of clinical trials (“Clinical Trials”) are key research
tools for advancing medical knowledge, utilizing innovative strategies to improve patient care. Clinicians
conduct these Clinical Trials in specific patient populations to determine whether an investigational
medicine works and is safe to use in patients. Participation in U.S. Food and Drug Administration (“FDA”)
or other relevant regulatory agency accepted Clinical Trials is the best way to access investigational
therapies. As such, at this time, participation in a Clinical Trial is the only way for patients to gain access
to the investigational therapy, Cusatuzumab, under development by Oncoverity.
We recognize that not all patients will meet the eligibility requirements for participation in a Clinical
Trial and as a result, understand that some may seek pre‐approval access when no other comparable
therapy is available for use, also referred to as expanded access, compassionate use, or named-patient
use (21 CFR part 312, subpart I).
Oncoverity relies on guidelines provided by the FDA and other regulatory agencies along with other
important factors when evaluating our ability to provide pre‐approval access for Cusatuzumab. Key
considerations include:
1) Whether there is sufficient evidence that the potential benefits to the patients outweigh the
potential risks, based on available safety and efficacy data
2) Whether sufficient clinical data is available to identify a recommended dose that might be
effective and is reasonably safe
3) Whether providing pre‐approval access outside of a Clinical Trial setting will compromise, delay,
or otherwise have a negative impact on ongoing Clinical Trials and/or future access by all eligible
patients
4) Whether there is adequate supply of product (Cusatuzumab) for Clinical Trials and any other
usage, such as expanded access
5) Whether it is feasible to provide any necessary follow up outside of the Clinical Trial setting
As we are about to embark on a Phase 2 study of Cusatuzumab, we are collecting additional safety and
efficacy data, which will be evaluated at the end of that study. Once that data is collected, we will reevaluate our policy on expanded access.
Here is the link to our open trial:
https://clinicaltrials.gov/study/NCT04023526?term=cusatuzumab&rank=1
At this time, we are not accepting requests for expanded access. If you have any additional questions
you may contact: helpdesk@oncoverity.com
Available Therapies via Single-Patient EA
Cusatuzumab currently being investigated in AML
Ondine Biomedical
Expected Application Timeframe
7 business daysSingle-Patient EA Policies/Criteria
Named Patient Access (Single Patient IND in the U.S.) – where a qualified healthcare professional requests an investigational medicine for use for a single patient because, in their judgement, currently available therapies are not satisfactory. Provision of the investigational medicine by Ondine may or may not require approval by FDA as well as an Institutional Review Board.
Available Therapies via Single-Patient EA
Non-antibiotic nasal decolonization. See https://www.ondinebio.com/solutions/steriwave/ . This is a light-based therapy that can decolonize the nose of bacteria, viruses and fungi within 4 minutes. Typically used for pre-operative patients as well as for vulnerable populations or immunocompromised patients.
Oneness Biotech Company Limited
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Expanded Access Policy
All expanded access programs must be conducted in agreement with applicable legal and regulatory requirements related to providing an investigational product under expanded access protocols. Oneness Biotech believes that participation in one of our clinical trials is the best way to access our investigational drug product. We encourage patients to speak with their physicians regarding participating in clinical trials. In exceptional cases where patients with serious diseases are unable to participate in clinical trials and have exhausted all available options, requests for EAP will be considered, on a case-by-case basis, if it meets the following criteria listed below:
Criteria for Consideration of a Request for EAP
1. A patient has a serious, life-threatening or chronically debilitating disease;
2. There is a clear understanding that the investigational product is intended to treat diabetic foot ulcers for which use is requested by the licensed physician and the patient;
3. The investigational product is regarded by the licensed physician and the patient that the available clinical data presents evidence of efficacy such that a clinically meaningful benefit may be expected;
4. There is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition;
5. The patients are ineligible for, or otherwise unable to participate in, a clinical trial for the product;
6. The treating physician must complete FDA Form 3926 and is willing to open a Treatment IND with the FDA, including file paperwork with FDA and IRB (for many expanded access request types), and is responsible for patient care and reporting;
7. The treating physician should provide the completed Form to Oneness Biotech for review prior to submission to FDA;
8. The treating physician and the patient (and the patient’s family or custodians) agree to waive claims for damages against Oneness Biotech;
9. There is adequate supply of the investigational product to meet all needs for patients enrolled in ongoing clinical studies, and that designating investigational product for an expanded access treatment use will not compromise supply or otherwise postpone providing the new treatment, once approved, to the broader patient population;
10. Providing expanded access to an investigational product shall not negatively jeopardize the initiation, conduct, or completion of clinical investigation(s) and the overall development program to support registration of the product.
Available Therapies via Single-Patient EA
ON101 (under IND 079526) for indication of diabetic foot ulcers
Orano Med
Single-Patient EA Policies/Criteria
Currently, RadioMedix, Inc. and Orano Med, do not offer an expanded access program and are not accepting expanded access requests for AlphaMedix™. Our development resources are focused on conducting clinical trials that evaluate the safety and efficacy of our treatment.
Orinove Inc.
Expected Application Timeframe
2 weeksSingle-Patient EA Policies/Criteria
Orinove Inc. is committed to developing novel and selective medicines to patients with serious or life-threatening conditions.
We are focused on enrolling and conducting the clinical trials necessary to gain regulatory approval to make our medicines available broadly to patients as quickly as possible. Orinove believes that participation in our clinical trial is the most appropriate way to access our investigational product therefore, at this time, Orinove is not making its investigational product available on expanded access.
We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy. In line with the 21st Century Cures Act, Orinove may revise this policy at any time.
If you have questions about our investigational product or expanded access, please contact wangweiai1@orinove.com. Orinove anticipates that it will acknowledge receipt of any expanded access questions or requests within two weeks of receipt.
For active clinical trials with Orinove’s investigational agents, please search “Orinove” at www.clinicaltrials.gov.
Orphazyme
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
In general, Orphazyme will consider granting access to an investigational drug on a case-by-case basis if an Early Access Program is open in the country of residence of the patient and the following criteria are met:
• The patient has a serious or life-threatening medical condition within the disease area and the patient population that Orphazyme is investigating and planning to pursue approval by the national regulatory authorities.
• The patient has no access to a suitable approved treatment for the disease condition or no adequate treatments are available.
• The patient does not qualify to participate in any ongoing or planned clinical trials in a reasonably accessible geographical location.
• The patient has a disease for which there is sufficient clinical evidence of benefit from the use of the investigational drug, and the benefits likely outweigh the known or anticipated risks.
• Providing Early Access does not in any way disrupt the clinical development program and/or the regulatory pathway towards marketing authorization leading to broader access.
• There is an adequate and sustainable supply of the investigational drug, beyond what is required for all ongoing and planned clinical trials.
• The request for Early Access is received from a licensed physician with expertise in treating the disease and understands the potential risks and benefits of the investigational drug.
• The requesting physician agrees to comply with Orphazyme’s Early Access Program specifications as well as with the applicable laws and regulations governing the use of the investigational drug. Early Access
ends once the investigational drug is commercially available or reimbursed in a country. If Orphazyme decides to discontinue clinical development of the investigational drug for a specific disease area, Early Access will be discontinued.
Available Therapies via Single-Patient EA
Arimoclomol for Niemann-Pick type C (NPC)
Disease/Category-Specific EA Policies/Criteria
Otsuka America Pharmaceutical
Expected Application Timeframe
1 weekPacylex Pharmaceuticals, Inc.
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
At this time, based on the evidence generated to date, Pacylex has decided not to offer Expanded Access (EA) to any of our investigational products and will not approve EA requests. We will re-evaluate the status of our policy based on data generated from ongoing and future clinical trials.
This will be re-evaluated based on the criteria listed below.
3.1 Criteria
Where there is a possible need for patients to gain early access to a new medicine that is not generally available for prescription, Pacylex will consider the need for early access using the following criteria:
- Where it is expected that there is a need for ongoing treatment with one of our new medicines, we recognize that this must be through normal health services once the new medicine is available for prescription. As such we will only conduct clinical studies and provide early access outside of studies, in those countries where we intend to make the medicine available through normal prescription channels after its approval.
- We recognize that we must have a minimum level of evidence that the new medicine is likely to work before considering early access and must not provide excessive risk to any patients who may receive the new medicine early.
- While addressing this patient need, we will ensure that if we agree to provide early access to our new medicines, that there is sufficient medicine available to ensure the supply will not run out for those patients receiving it early, or for those patients in our clinical studies. This is because we want to ensure that when providing early access, it does not prevent us completing clinical studies and regulatory approval that would lead to wider access through normal routes of prescription.
- Any early access programs we provide will be in line with the laws and requirements of the country involved.
3.2 Continued treatment after clinical studies
To be sure that our new medicines will work effectively and be safe enough to be prescribed for the diseases patients are suffering from, we study them rigorously to see whether they provide adequate relief. The results of these clinical studies allow the regulatory authorities to independently assess whether to allow the new medicines to be prescribed. As this can take several years, patients who participate in our clinical studies may have to wait for a new medicine to become available on prescription.
Where there may be a benefit for a patient to continue to be treated after the clinical study has ended, but before it is available on prescription, we will decide if we have enough information to justify early access. This will depend on the severity of the disease we are studying, how much evidence we have that the medicine works as expected and is sufficiently safe, and whether alternative effective medicines are already available on prescription. If we plan for continued treatment after a clinical study, we will include this information in our study documents so that it is clear to each patient whether this is a possibility before they start in the study.
We understand that it can be difficult to decide to participate in a clinical study, so will ensure we make this information as clear as possible. To be sure we can assess whether the new medicine continues to work effectively and is safe enough to continue to be used during early access, we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.
3.3 Compassionate or emergency use outside of clinical studies
The best way to see if a new medicine is sufficiently safe and effective is to run clinical studies. As such we prefer that, where possible, patients can participate in our clinical studies. We recognize however that not everyone has this opportunity. Where patients who cannot enroll in a clinical study are suffering from serious or immediately life-threatening diseases, and there are no satisfactory alternative treatments available, we will consider early access if there is enough evidence available in their condition (as described above) and whether there is a possibility of them being able to access the medicine on prescription in the future.
In the same way as after a clinical study, we want to be sure we can assess whether the new medicine continues to work and is safe enough to continue to be used. As such we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.
3.4 Discontinuation of early access
When a new medicine receives approval from the regulatory authorities for the disease we are studying, and is available for prescription in the patient’s country, we will phase out the early access to allow the health system to prescribe the new medicine in the normal manner.
If, however, the medicine is approved but not available for prescription for some reason, and there is no alternative medicine available, we will work with country authorities to discuss if we can jointly set up a patient-assistance program to allow patients who are benefitting from it, who do not have alternative treatment options, to continue to receive the new medicine. Patient safety is our primary concern, and we respect patient safety by continually evaluating all the evidence that we have available to ensure that early access use of new medicines remains effective and appropriately safe. If, however, the evidence demonstrates that the new medicine is not going to be as sufficiently effective or safe as we had hoped, we will discontinue all early access and work with patients and their physicians to help them safely transition patients to other treatments. When regulatory authorities do not approve the use of the new medicine for prescription, because it is considered insufficiently safe or effective, and Pacylex discontinues its work to achieve regulatory approval, we will work with patients and their physicians to help them safely transition patients to other treatments.
Available Therapies via Single-Patient EA
PCLX-001 - Diffuse Large B-Cell Lymphoma (DLBCL) and Acute Myeloid Leukemia (AML)
Pardes Biosciences, Inc.
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
N/A – EA unavailable at this time
Available Therapies via Single-Patient EA
N/A – EA unavailable at this time
Parion Sciences, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
We are unable to offer single patient expanded access at this time.
PeproMene Bio Inc.
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
PeproMene Bio Inc. is committed to developing safe and effective immune therapies such as BAFFR CAR-T cells to improve the lives of patients with cancer and immune disorders.
At this time, PeproMene does not offer an Expanded Access program. Our lead product, PMB-CT01 (BAFFR-CAR T cells) is investigational, which means that it has not been approved by regulatory health authorities, such as the United States Food and Drug Administration (FDA). While we fully understand that there may be desire to access to our products and recognize the significance of Expanded Access programs, our current priority is to advance our clinical trials as efficiently as possible to ultimately benefit the greatest number of patients. Therefore, PeproMene believes that participation in one of our clinical trials is the best way to access our investigational BAFFR-CAR T therapy. We encourage patients to speak with their physicians regarding participating in the clinical trials. Information on PeproMene’s ongoing clinical trials (NCT05370430 and NCT04690595) can be found in www.clinicaltrials.gov.
As more clinical data on the safety and efficacy of our investigational drugs become available, PeproMene will review and update its policy on Expanded Access. If you have questions about this policy, please contact info@pepromenebio.com.
Pfizer
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Pharnext SAS
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
ACCESS TO PHARNEXT INVESTIGATIONAL MEDICINES PRIOR TO REGULATORY APPROVAL
(FOR US PATIENTS ONLY)
Pharnext develops innovative new medicines designed to address unmet medical needs of patients in major therapeutic areas. We are focused on creating new medicines that address important, real world concerns of patients and their families. We currently have investigational treatments in clinical trials for a number of different diseases, as described in the PRODUCTS section of our website at https://www.pharnext.com/en/products/r-d-pipeline
Pharnext’s goal is to efficiently bring safe and effective medicines to all appropriate patients by conducting rigorous clinical trials and obtaining marketing approval by regulatory authorities, including the U.S. Food and Drug Administration (FDA). Prior to such approval, participation in one of our clinical trials is the optimal way for patients to gain access to our investigational medicines. However, we understand that some patients may not be able to participate in a clinical trial and may seek access to investigational treatments before they are approved. These situations are often referred to in various ways, including expanded access, early access, pre-approval access, compassionate use and emergency use.
If early data from clinical trials suggest that an investigational treatment can offer benefits for patients facing serious or life-threatening conditions, Pharnext may consider requests to provide access outside of a clinical trial. Our top priority when evaluating requests for expanded access to investigational medicines is to ensure that we are not putting patients at risk of unnecessary harm.
Pharnext will consider an expanded access program, or a single request for expanded access of an investigational medicine, only if all of the following criteria are met:
• The patient’s licensed physician submits an unsolicited request for expanded access
• The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy
• There are no comparable or satisfactory alternative therapies approved and available to treat the disease or condition
• There is preliminary clinical evidence of effectiveness and acceptable safety of the investigational treatment for that patient’s condition
• The potential benefit of the investigational medicine to the patients is greater than the potential risk
• Staff and other required resources are available to provide the product and there is an adequate supply of the investigational medicine
• Providing the investigational medicine will not interfere with the timely completion of clinical trials that could lead to marketing approval and thereby could delay its availability to other patients
• Expanded access will only occur in a country where Pharnext expects to file for regulatory approval of the product
Patients interested in obtaining access to a Pharnext investigational therapy must do so via their physician. Physicians should contact Pharnext Medical Information Center at medical@pharnext.com. All requests will be carefully considered, and a written or oral response will be provided to the treating physician within two weeks of receiving a request. For further information on available expanded access programs, visit www.clinicaltrials.gov and search ‘expanded access programs’.
Available Therapies via Single-Patient EA
NA
Disease/Category-Specific EA Policies/Criteria
NA - see above
Pharvaris
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Pharvaris is committed to serving patients’ health needs by developing safe and effective therapies and making them available for use as quickly as possible. We believe that controlled clinical trials provide the best path for Pharvaris to generate the safety and efficacy information needed to obtain regulatory approval so that we can make our medicines available to all patients.
Pharvaris always seeks to provide the quickest and broadest access to its medicines, balancing benefit and risk to all patients. We support the principle of expanded-access programs consistent with the best standards for safe and effective use of an investigational therapy, as well as our ability to logistically provide a product. Pharvaris will consider requests for expanded access from qualified physicians, balancing multiple factors.
When evaluating potential use of an investigational therapy, we will consider several factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ guidelines including both patient and scientific perspectives. Eligibility requirements include but are not limited to:
- Sufficient scientific evidence to support both the safety and the efficacy of the compound’s mechanism for this indication
- A serious or life-threatening illness, with no other satisfactory treatment options (such as approved products or enrolling clinical trials)
- Sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on all available safety and efficacy information
- Support that providing the investigational drug will not interfere with clinical trials that could support the investigational drug’s development or regulatory approval for the treatment indication, which could affect future patient access
- The evaluation of these, and other, questions may require additional investigation or interaction with the requesting physician, other clinical investigators, or regulatory authorities. Favorable answers alone provide no guarantee that Pharvaris will be able to provide pre-approval access to an experimental medicine.
Requests for access to investigational therapies must be made by a qualified and licensed physician to Pharvaris (expandedaccess@pharvaris.com) and will be evaluated by a core team. Pharvaris anticipates it will acknowledge receipt of such requests within five business days after receipt of an inquiry.
Pieris Pharmaceuticals Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
At Pieris Pharmaceuticals Inc., we are committed to developing novel medicines to improve the clinical outcomes of patients suffering from cancer and respiratory diseases. Cinrebafusp alfa (PRS-343) is in clinical development and currently being evaluated as a treatment for gastric cancer.
We understand the interest of patients in accessing cinrebafusp alfa outside of clinical trials and prior to regulatory approval. However, participation in one of our clinical trials is currently the most appropriate way to access our investigational therapies. Pieris does not offer cinrebafusp alfa via expanded access at this time.
We recognize the need for Expanded Access programs, and we will re-evaluate the status of our policy based on data from ongoing and future clinical trials.
If you have additional questions, please consult your physician or to learn more about our clinical programs, including eligibility criteria and locations to visit www.clinicaltrials.gov.
PolyPid Ltd
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
There are seriously ill patients who will not be eligible for our clinical trials and may be in a high risk to experience an infection post a surgical intervention. In these circumstances, POLYPID will consider providing the requesting surgeon with pre-approved access to D-PLEX, for the prevention of infection of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:
• The patient is scheduled for an abdominal or a median sternotomy surgery for any reason;
• The patient is not eligible to participate in POLYPID clinical trials;
• The patient has a serious or life-threatening illness or condition and his/her likelihood to develop a serious infection due to surgical intervention is high;
• The patient has a disease or condition for which there is sufficient evidence of a projected benefit from the use of the D-PLEX and the benefit outweighs the known or anticipated risks;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting surgeon’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or a regulatory review or an approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available and can be supplied in the appropriate timeline of request.
Available Therapies via Single-Patient EA
D-PLEX, a new formulation of Doxycycline for local administration that is intended for prevention of Surgical Site Infections (SSIs) post cardiac surgery with median sternotomy and post abdominal surgery.
Disease/Category-Specific EA Policies/Criteria
https://www.polypid.com/wp-content/uploads/2020/08/PolyPid-Expanded-Access-Policy-FINAL.pdf
Poxel
Expected Application Timeframe
1 monthSingle-Patient EA Policies/Criteria
Patient Eligibility Criteria:
To be eligible for access to an investigational product, a patient must meet the following criteria:
- Be an adult male with a confirmed diagnosis of X-linked adrenoleukodystrophy.
- Have undergone standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
- Are not eligible or able to participate in any planned or ongoing clinical trial of the investigational product(s).
- Has manifestations of disease for which there is sufficient evidence of a projected benefit (e.g. based on prior positive clinical trial results) from the use of the investigational product and the benefit outweighs the known or anticipated risks.
- Any other pertinent medical criteria for access to the investigational product, as established by Poxel.
- Investigational Product Criteria
In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
- The product is in active clinical development.
- There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
- The provision of the investigational product will not interfere with or compromise the clinical development of the product, and that there is sufficient investigational product available to ensure that supply constraints will not impede continued use of the product(s) by patient(s) or the conduct of our clinical trials.
Treating Physician Criteria and Responsibilities:
The physician(s) attending to the patient(s) who is/are receiving an investigational product through expanded use access is (are) properly licensed in the U.S. and fully qualified to administer the product. The physician must agree in writing to comply with:
- Any applicable legal and regulatory requirements related to providing an investigational product under Expanded Access.
- Any Poxel requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property.
Available Therapies via Single-Patient EA
PXL065 for an adult male with a confirmed diagnosis of X-linked adrenoleukodystrophy.
Disease/Category-Specific EA Policies/Criteria
https://www.poxelpharma.com/en_us/pipeline/clinical-trials/expanded-access-policy
Prestige Biopharma
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
At Prestige Biopharma, we are committed to developing safe and effective drugs to improve the health and well-being of patients. We recognize that some patients may have serious or life-threatening conditions and are not able to participate in our clinical trials. For these patients, Expanded Access to investigational drugs may be an option.
Currently, our investigational medicines are in early phase development, and we are not yet providing Expanded Access. We believe that participating in our clinical trials is the best way for patients to potentially access our investigational medicines prior to regulatory approval. We consider this approach to be the best opportunity to bring safe and effective treatments to patients. We encourage any person interested in gaining access to our investigational medicines to consult their physician regarding the possibility of participating in one of our clinical trials. Please review the information about Prestige Biopharma’s ongoing clinical trials found on this website as well as at https://clinicaltrials.gov/.
We wish to assure patients that we are constantly evaluating our policies and procedures. As such, Expanded Access may be reassessed in the future as our investigational medicines advance further in clinical trials and more data to support the safety and effectiveness becomes available.
If you have any questions about our Expanded Access policy or our clinical trials, please contact us at info@prestigebio.com.
PRG Science and Technology Co., Ltd.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
At PRG S&T, we commit to bringing new, innovative therapies to patients by conducting rigorous clinical trials and obtaining marketing approval by the FDA and other regulatory authorities. At this point of time, PRG S&T believes that participation in one of our clinical trials is the most appropriate way to access our investigational medicine. However, we may also provide our investigational medicine for use through expanded access program.
Requests for expanded access must be made by a physician on behalf of their patient. The eligibility for a patient to participate in the expanded access program will be reviewed and evaluated based on the patient’s medical information. The request for access to a PRG S&T investigational medicine will only be considered if the patient's treating physician is committed to, and supportive of, the requested treatment.
Questions with regards to the expanded access to PRG S&T investigational medicine may be sent to us via the contact information listed on our website. Should the circumstance permits change to our expanded assess policy, updates will be posted to this website.
Available Therapies via Single-Patient EA
Progerinin for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS) and Werner Syndrome (WS)
Pulmocide
Single-Patient EA Policies/Criteria
The goal of our current clinical study program for PC945 is to enroll patients and obtain clinical data that is required by the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other regulatory authorities for review and approval of marketing applications for this investigational agent. We believe that focusing on clinical studies is the most appropriate way to achieve this goal; therefore, we do not currently offer access to PC945 outside of clinical studies and, at this time have no Expanded Access program (sometimes referred to as “compassionate use” or “pre-approval access”).
Purdue Pharma L.P.
Expected Application Timeframe
N/ARactigen Therapeutics
Expected Application Timeframe
10 Business DaysSingle-Patient EA Policies/Criteria
Ractigen Therapeutics is at the forefront of developing small activating RNA(saRNA) drugs utilizing the RNA activation (RNAa) mechanism to up-regulate endogenous gene expression. RAG-01 is a pioneering saRNA drug engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. It is currently being investigated in a phase Ⅰ, open label, multi-center study in patients with non-muscle-invasive bladder cancer (NMIBC) who have failed Bacillus Calmette-Guérin (BCG) therapy. We are conducting the clinical trial aiming at gaining regulatory approval to make RAG-01 available to all eligible patients as quickly as possible.
Ractigen is not currently making its investigational products available on an expanded access basis anywhere in the world, as we believe that our clinical trials are the most appropriate way to access our investigational products. We encourage patients who are interested in accessing RAG-01 treatment to talk to their physician about participating in a clinical trial.
If we determine that expanded access to RAG-01 becomes appropriate in the future, this policy will be updated. If you have further questions, please feel free to contact us at expanded.access@ractigen.com with any questions.
Consistent with the 21st Century Cures Act, Ractigen Therapeutics may revise this policy at any time.
RadioMedix
Single-Patient EA Policies/Criteria
Currently, RadioMedix, Inc. and Orano Med, do not offer an expanded access program and are not accepting expanded access requests for AlphaMedix™. Our development resources are focused on conducting clinical trials that evaluate the safety and efficacy of our treatment.
Rapa Therapeutics, LLC
Phone Number & Email
Single-Patient EA Policies/Criteria
We do not plan to offer Expanded Access for our studies.
Recursion Pharmaceuticals, Inc.
Single-Patient EA Policies/Criteria
At Recursion Pharmaceuticals, we are committed to radically improving the lives of patients and their families by developing therapies that will make a lasting, positive, and transformative impact on diseases and conditions for which there are high unmet needs.
We believe that the best way to succeed in our mission is through well-designed clinical trials that determine the safety and effectiveness of investigational medicines. We understand that in some rare and specific circumstances, when enrollment into a clinical trial is not possible, physicians caring for patients with serious or life-threatening conditions or diseases may seek special access to investigational medicines. When appropriate and through an expanded access protocol, Recursion will consider individual Expanded Access, sometimes called “Compassionate Use” or “Early Access”, requests for an investigational medicine outside of an ongoing clinical trial for the indication being investigated by the company.
There may be additional requirements, but to start, a licensed treating physician must submit the request for Expanded Access in writing by contacting ExpandedAccess@Recursion.com. The physician must be able and willing to obtain a treatment IND, or regional equivalent, and Institutional Review Board or Ethics Committee approval, for the patient prior to initiating treatment with our investigational medicine.
We will consider applications to provide the investigational drug that will not, among other things, compromise the scientific validity of our broader development programs, or interfere with or delay clinical trials or regulatory filings designed to make the drug available to a larger patient population.
We endeavor to respond to Expanded Access requests within one week after the steps for submission are completed.
It is important to remember that investigational medicinal products have not yet received regulatory approval; thus, their potential risks and benefits are not yet established. Physicians and patients should consider all possible benefits and risks when seeking access to an investigational medicinal product. For information on available clinical trials visit: www.ClinicalTrials.gov.
cts have not yet received regulatory approval;
thus, their potential risks and benefits are not yet established. Physicians and patients should consider all
possible benefits and risks when seeking access to an investigational medicinal product. For information on
available clinical trials visit:
Relmada Therapeutics
Expected Application Timeframe
N/AAvailable Therapies via Single-Patient EA
To provide expanded access of REL-1017 to patients with MDD who do not have access to other effective therapy and are not eligible for other clinical trials with REL-1017.
Inclusion Criteria:
• Patients with Major Depressive Disorder who may potentially benefit from treatment with REL-1017 as determined by the treating physician.
• Have undergone appropriate standard treatments without success and in the opinion of the treating physician no comparable or satisfactory
alternative treatment is available to treat the disease or condition.
• Are ineligible or unable to participate in any ongoing clinical study of the investigational product.
• Condition for which, in the opinion of the treating physician, there is sufficient evidence of a potential benefit from the use of the investigational product, and that potential benefit outweighs the known or anticipated
risks.
• There is adequate information to support appropriate dosing for special population patients such as pediatric, elderly, renal or hepatic disease, etc.
Exclusion Criteria:
• History or presence of clinically significant health conditions which in the opinion of the Investigator would negatively impact the safety of the participant.
• History of allergy or hypersensitivity to methadone or related drugs.
• Pregnant or planning to become pregnant.
• Breast-feeding or planning to breast-feed.
RemeGen Co., LTD.
Expected Application Timeframe
7 DaysSingle-Patient EA Policies/Criteria
The following criteria are typically required before RemeGen would consider patients eligible for expanded access to a RemeGen investigational product. All criteria are subject to local laws and regulations.
• The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
• The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
• The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
• The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive which typically occurs when RemeGen has decided to seek marketing approval in at least one major market globally.
• RemeGen has decided to seek marketing approval in at least one major market globally.
• The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
• The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.
RemeGen cannot approve expanded access use requests in cases where doing so would violate company policy, local laws or regulations, or if access would present an unacceptable benefit/risk profile, as deemed by medical personnel of the company.
We commit to a careful and fair evaluation of each request by the appropriate medical experts at RemeGen within the boundaries of local laws and regulations. All requests for expanded access (typically made by the treating physician) will be acknowledged by medical personnel from RemeGen (or our agent) within 7 business days. Should the request be approved, the requestor must enter into an Expanded Access Agreement with RemeGen.
Available Therapies via Single-Patient EA
RC18 (telitacicept) for primary Sjögren’s syndrome
RemeGen Co., LTD.
Single-Patient EA Policies/Criteria
RemeGen Co., Ltd. is a leading biopharmaceutical company in China dedicated to fulfilling unmet medical needs for patients with life-threatening conditions. RemeGen Co., Ltd.'s main focus is research and development, manufacturing and commercialization of novel biologics, most notably monoclonal antibodies (mAb) , fusion protein and antibody-drug conjugates (ADCs). Headquartered in Yantai, Shandong Province, RemeGen Co., Ltd. has laboratory research facilities and offices in Beijing, Shanghai, Maryland and California.
Consistent with RemeGen's mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing.
We currently have a early-stage study for Platinum-Resistant Recurrent Epithelial Ovarian, Fallopian Tube and Primary Peritoneal Cancer. Our goal is to provide access to our medicine at the appropriate time and in a manner that is most beneficial to the relevant patient population. We believe enrollment in our ongoing clinical trials is the safest and most effective way of achieving this goal. We do recognize that some patients will not be eligible for our clinical trials and may wish to access our products through expanded access. However, at this time, we do not have the resources available to offer expanded access use of our investigational medicines. We encourage all patients and physicians who are interested in accessing our investigational medicine to visit the clinical trial https://clinicaltrials.gov to find out about enrolling in clinical trials.
Available Therapies via Single-Patient EA
RC88 monotherapy in patients with Platinum-Resistant Recurrent Epithelial Ovarian, Fallopian Tube and Primary Peritoneal Cancer
RemeGen Co., LTD.
Expected Application Timeframe
7 daysSingle-Patient EA Policies/Criteria
Consistent with RemeGen’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing. At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, RemeGen will consider providing a requesting physician with pre-approval access to a specific RemeGen investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.
The following criteria are typically required before RemeGen would consider patients eligible for expanded access to a RemeGen investigational product. All criteria are subject to local laws and regulations.
• The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
• The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
• The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
• The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive which typically occurs when RemeGen has decided to seek marketing approval in at least one major market globally.
• RemeGen has decided to seek marketing approval in at least one major market globally.
• The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
• The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.
RemeGen cannot approve expanded access use requests in cases where doing so would violate company policy, local laws or regulations, or if access would present an unacceptable benefit/risk profile, as deemed by medical personnel of the company.
We commit to a careful and fair evaluation of each request by the appropriate medical experts at RemeGen within the boundaries of local laws and regulations. All requests for expanded access (typically made by the treating physician) will be acknowledged by medical personnel from RemeGen (or our agent) within 7 business days. Should the request be approved, the requestor must enter into an Expanded Access Agreement with RemeGen.
http://www.remegen.com/index.php?v=show&cid=113&id=1006
Available Therapies via Single-Patient EA
Telitacicept for patients with Generalized Myasthenia Gravis
RemeGen, Ltd.
Expected Application Timeframe
7 daysSingle-Patient EA Policies/Criteria
The following criteria are typically required before RemeGen would consider patients eligible for expanded access to a RemeGen investigational product. All criteria are subject to local laws and regulations.
• The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
• The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
• The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
• The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive which typically occurs when RemeGen has decided to seek marketing approval in at least one major market globally.
• RemeGen has decided to seek marketing approval in at least one major market globally.
• The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
• The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.
Available Therapies via Single-Patient EA
Telitacicept for patients with moderate or severe SLE
Reneo Pharmaceuticals Inc.
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
Reneo is committed to developing promising new therapies to address the unmet medical needs of patients suffering from rare and seriously debilitating diseases. We currently have a late-stage clinical trial planned for the treatment of Primary Mitochondrial Myopathies (PMM) and early-stage studies for Glycogen Storage Disorder V (GSDV, also known as McArdle Disease) and Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD). Our goal is to provide access to our medicines at the appropriate time and in a manner that is most beneficial to the relevant patient population. We believe enrollment in our ongoing clinical trials is the safest and most effective way of achieving this goal. We do recognize that some patients will not be eligible for our clinical trials and may wish to access our products through expanded access. However, at this time, we do not have the resources available to offer expanded access use of our investigational medicines. We encourage all patients and physicians who are interested in accessing our investigational medicines to visit the clinical trial section below to find out about enrolling in clinical trials.
Available Therapies via Single-Patient EA
N/A
Renibus Pharmaceuticals
Expected Application Timeframe
10 business daysSingle-Patient EA Policies/Criteria
Renibus Therapeutics will evaluate and respond to a request that it receives on a case-by-case basis.
Available Therapies via Single-Patient EA
RBT-9 for the treatment of COVID-19 at the early stage (Stage I) of infection in patients who are at high risk for disease progression.
Sanofi
Expected Application Timeframe
1 business daySavara Inc.
Expected Application Timeframe
5 business daysAvailable Therapies via Single-Patient EA
Procedure for Submitting Requests to Savara:
Savara will consider expanded access requests from treating physicians subject to US laws and regulations. All requests should be submitted via e-mail to EAP.US@savarapharma.com.
Process for Review of Requests:
Savara is committed to a fair and impartial evaluation of each request for access to its investigational products. Therefore, all decisions are based solely on clinical circumstances and are guided by the criteria outlined below. Patients will be referred to ongoing clinical trials as the primary way to access investigational products.
When evaluating requests for expanded access, Savara considers all of the following criteria:
1. The patient for whom expanded access is requested suffers from a disease or condition that is serious or life-threatening.
2. There are no comparable or satisfactory alternative therapies or clinical trials available.
3. Sufficient preliminary efficacy and safety data exist to support an assessment that the benefit for the patient outweighs the potential risks and that the potential risks are not unreasonable in the context of the disease or condition being treated.
4. Sufficient clinical data are available to identify an appropriate dose (amount and frequency) of the investigational drug.
5. There is adequate drug supply to support the ongoing and necessary clinical trials as well as to support approved expanded access in a sustainable and equitable manner, until and if product becomes commercially available.
6. The patient is not eligible to participate in any ongoing clinical trials of the investigational drug.
7. Expanded access will not adversely affect the clinical development program, in particular, the initiation, conduct, or completion of the clinical trials that are required for regulatory approval.
8. The unsolicited request is made by a U.S. qualified and licensed physician who will take primary responsibility for supervising use of the investigational product from Savara and will comply with all applicable FDA regulatory requirements associated with treatment and use of an investigational product.
9. All required regulatory and institutional approvals have been obtained. The patient must provide written informed consent.
Requests for expanded access will be individually reviewed in accordance with these criteria. Savara is committed to evaluating all requests for expanded access in a fair and equitable manner. All requests will be evaluated by medical professionals and decisions will be based on available scientific evidence at the time of the request.
Questions regarding Savara's ongoing U.S. expanded access program(s) can be forwarded to: EAP.US@savarapharma.com
This policy is not applicable to countries outside the U.S. If you have questions on expanded access in a non-US country, please submit your request to info@savarapharma.com.
Scilex Pharmaceuticals, Inc.
Single-Patient EA Policies/Criteria
Scilex Holding Company (Scilex) is dedicated to the development and commercialization of non-opioid pain management products using innovative delivery technologies and is actively evaluating the safety and effectiveness of these therapies.
Expanded Access Policy
The US Food and Drug Administration (FDA) has established criteria for the provision of investigational medicines* to patients outside of clinical trials (compassionate use/expanded access). That guidance provides that expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medicine for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available(1).
Expanded access may be appropriate when the following factors have been considered(2):
- Patient has a serious or life-threatening illness or condition and is not responsive to or able to tolerate any available treatment option
- The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition
- Patient enrollment in a clinical trial is not possible
- Potential patient benefit justifies the potential risks of treatment
- Providing the investigational medicine will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational medicine for broader patient access
- Adequate supply of the investigational medicine is available
Currently, Scilex is not accepting applications for expanded access of our investigational medicines. Our development resources are focused on conducting clinical trials that evaluate the safety and effectiveness of our investigational medicines for treating acute and chronic pain. Our clinical trial programs are the primary way to gain access to a Scilex investigational medicine and Scilex encourages patients to speak with their treating physicians about participating in a clinical trial whenever possible.
Scilex may reconsider making its investigational medicines available through an Expanded Access Program in the future as we continually evaluate each of our investigational medicines for appropriateness of expanded access based on FDA’s established criteria.
Healthcare providers, patients and caregivers interested in learning more about Scilex’s investigational therapies should contact Scilex at eap@scilexpharma.com.
Additional information regarding our clinical trials may be accessed on www.clinicaltrials.gov.
*This policy is subject to change. Scilex may revise this policy at any time and may establish different expanded access policies for each investigational medicine under development. The posting of this policy by Scilex shall not serve as a guarantee of access to any specific investigational medicine by any individual patient.
**Investigational medicines have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medicine may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.
References:
(1) https://www.fda.gov/news-events/public-health-focus/expanded-access (accessed September 1, 2022); see also 21 C.F.R. 312.305(a)
(2) https://navigator.reaganudall.org/companies-and-sponsors#introduction (accessed September 1, 2022)
Scorpion Therapeutics, Inc.
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
Our EAP policy is described below:
Several factors consistent with the FDA and other regulatory agencies’ guidelines, are essential when considering expanded access requests:
• The illness must be serious or life-threatening with no other satisfactory treatment options (such as approved products or open clinical trials)
• There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on available safety and efficacy information
• The ability to provide a therapy in a fair and equitable manner, so that there is adequate manufacturing capacity to provide therapies across all ongoing clinical trials and expanded access programs
• Whether granting expanded access would potentially compromise the scientific validity of broader development programs, or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients
Given the early stage of our development program, we believe that participation in our clinical trial is the most appropriate way to access our investigational therapy. Therefore, we are not currently making our investigational product available through an expanded access program. If you have additional questions, please speak with your physician, or have your physician contact clinicaltrials@scorpiontx.com.
In line with the 21st Century Cures Act, Scorpion Therapeutics, Inc may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on clinicaltrials.gov if such record becomes active.
SCYNEXIS, Inc.
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
SCYNEXIS is committed to making Investigational Products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the SCYNEXIS requirements, may request information about how to apply for access to SCYNEXIS’ Investigational Products by contacting the company.
Available Therapies via Single-Patient EA
SCY-078 is a triterpenoid, glucan synthase inhibitor, antifungal agent undergoing investigation for:
• The treatment of invasive candidiasis, including candidemia (IC).
• The treatment of invasive aspergillosis (IA), including IA that is refractory to or intolerant of other antifungal therapies.
• The treatment of invasive candidiasis in patients who are refractory to or intolerant of other antifungal therapies.
Scytek Laboratories Inc
Single-Patient EA Policies/Criteria
Scytek Laboratories Inc- #1 diagnostic reagents: Special Stains, Antibodies, Microbiology ScyTek Biotech Life Sciences manufactures diagnostic reagents, Special Stains, Antibodies, Hematology, Immunohistochemistry, buffers, Microbiology, Hematoxlyn, Gram Stain kit, Methylene Blue Solution, Giemsa Stain kit etc
https://www.scytek.com
Serac Healthcare
Expected Application Timeframe
5 DaysSingle-Patient EA Policies/Criteria
Serac Healthcare will consider providing a requesting physician with pre-approval access to the Serac Healthcare investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.
- The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks
- The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition
- Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access
- Adequate supply of the investigational drug is available
- No viable alternative option is available
Available Therapies via Single-Patient EA
Maraciclatide is an in vivo imaging agent (non-therapeutic) for use in endometriosis.
Servier Pharmaceuticals
Expected Application Timeframe
1 business daySingle-Patient EA Policies/Criteria
Single-Patient Expanded Access Policies and Criteria -. Physicians seeking general information may contact Servier by emailing expandedaccess@servier.com .Available Therapies via Single-Patient EA
Vorasidenib - The vorasidenib EAP is designed to provide access to vorasidenib for patients with IDH1- or IDH2-mutated glioma who are not eligible for other vorasidenib clinical trials, and who in the opinion of the treating oncologist would potentially benefit from treatment from vorasidenib.Disease/Category-Specific EA Policies/Criteria
https://www.earlyaccesscare.com/companies/servier-pharmaceuticals-llcVoShanghai Henlius Biotech, Inc
Single-Patient EA Policies/Criteria
Henlius is a global biopharmaceutical company with the vision to offer high-quality, affordable, and innovative biologic medicines for patients worldwide with a focus on oncology, autoimmune diseases, and ophthalmic diseases.
HLX42, a novel antibody-drug conjugate (ADC) that targeting epidermal growth factor receptor (EGFR),is one of the first ADC candidates of Henlius to enter into clinical development. HLX42 is designed to overcome the resistance of current EGFR-targeted therapies and to provide more effective treatment options to fulfill the unmet clinical needs. HLX42 is to be investigated as a single agent in a Phase I trial for the treatment of solid tumors, including EGFR-mutated non-small cell lung cancer that progressed on a third-generation EGFR tyrosine kinase inhibitor.
Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational therapy for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Henlius understands the interest of patients in accessing HLX42 outside of clinical trials for potentially life-threatening diseases, however we do not provide any Expanded Access programs for HLX42 at this time.
Henlius recognizes the need for Expanded Access programs and will revise this policy based on data from future clinical trials.
Shanghai Miracogen Inc.
Available Therapies via Single-Patient EA
Shanghai Miracogen Inc. (Miracogen) is a biopharmaceutical company focusing on development of innovative antibody-drug conjugates (ADC) and bringing new therapies for patients with various cancers. Miracogen conducts human clinical trials for its investigational products to generate safety and efficacy data as required to support the regulatory approval by the U.S. Food and Drug Administration (FDA) and regulatory authorities in other countries and subsequent wider accessibility to patients.
Considering the stage of development of the investigational products, Miracogen is seeking to retain its capacity of manufacturing and drug supply to assure adequate supply to ongoing clinical trials and development programs to generate data on the safety and efficacy of the investigational products in order to support regulatory approval and make the products accessible to the broader patient population. At this time, Miracogen is unable to provide any of its investigational drug products on an expanded access or right-to-try basis. We encourage patients to speak with their physicians and to participate in the available clinical trials.
If you have any questions about our expanded access policy, please contact Miracogen via email at: expanded_access@miracogen.com.cn.
For more information on the available clinical trials, please visit https://clinicaltrials.gov and search company, condition, or treatment. For additional information on Expanded Access, please visit the U.S. Food and Drug Administration at: https://www.fda.gov/news-events/public-health-focus/expanded-access.
In line with the 21st Century Cures Act, Miracogen may revise this policy at any time.
Shanghai SIMR Biotechnology Co., Ltd
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
At SIMR we aspire to discover and develop differentiated therapeutics for the treatment of peripheral neuropathic pain. SIMR’s lead product, SR419, is a candidate drug with novel mechanism of action for the treatment of peripheral neuropathic pain, which is discovered and developed by SIMR. Peripheral neuropathic pain is the pain caused by damage or pathological changes of peripheral nerves, including post-herpetic neuralgia, diabetic peripheral neuropathy, chemotherapy-induced peripheral neuropathy, and postoperative peripheral neuropathy. The results of preclinical studies show that SR419 has excellent safety and pharmacokinetic properties. In addition, SR419 demonstrates the potential of better analgesic efficacy and fewer side effects than pregabalin, a commonly used first-line treatment for neuropathic pain. SR419 has completed phase I clinical trials in the Australia and China with positive safety results, and is planning to carry out international multi-center phase II clinical trials. At SIMR, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval, making SR419 available to patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access SR419 at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at www.clinicaltrials.gov. SIMR understands the interest of patients in accessing SR419 outside of clinical trials and prior to regulatory approval for potentially life threatening diseases, however we do not currently offer any Expanded Access programs for SR419. SIMR recognizes the need for Expanded Access programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.Disease/Category-Specific EA Policies/Criteria
http://www.simrbio.com/en/ExpandedAccessPolicy/Shire
Expected Application Timeframe
3 business daysSilence Therapeutics plc
Single-Patient EA Policies/Criteria
At Silence, our vision is to transform peoples’ lives around the world by silencing diseases through our precision engineered medicines and driving positive change for the communities around us. As pioneers in the design and development of siRNAs (short interfering RNAs), we are advancing a new generation of medicines to potentially address the needs of patients who have limited or inadequate treatment options. Our investigational product candidates are designed to harness the body's natural mechanism of RNA interference (RNAi) to precisely target and silence disease-associated genes.
Silence conducts clinical trials to ensure that our investigational product candidates are safe and effective, which supports approval of the treatment by regulatory authorities and ultimately enables patients to access approved, safe and effective treatments.
At this time, Silence does not offer an expanded access program for its investigational products and these can only be accessed by participation in a clinical trial. If in the future Silence decides to offer an expanded access program, Silence will update its policy accordingly. If you are a patient who is interested in accessing one of our investigational products by participating in a clinical trial, please consult with your physician. Anyone who wishes to learn more about Silence’s investigational products or this policy can contact Silence by emailing info@silence-therapeutics.com. More information on Silence’s clinical trials can be found at www.clinicaltrials.gov.
Silk Road Therapies, Inc.
Expected Application Timeframe
N/AAvailable Therapies via Single-Patient EA
At this time Silk Road Therapies, Inc./Ipekyolu Ilac Ltd. is testing a topical pentoxifylline formulation under US IND, but exclusively conducted in a trial in Istanbul, Turkey. At this time, we are unable to import our topical formulation from Turkey and do not have sufficient supply to offer expanded access to this drug. Therefore, until such time as the proof of concept trial is completed, Silk Road Therapies, Inc. is not offering expanded access beyond participation in the Istanbul trial.
However, we anticipate completing the proof of concept trial 3Q2019 and having reformulated material available in the US 1Q2020. At that time, we will revise this expanded access policy and may permit access to our product for patients who are ineligible for entrance into our later phase III pivotal trial.
SonALAsense, Inc.
Expected Application Timeframe
5 daysSingle-Patient EA Policies/Criteria
In very specific cases, SonALAsense may be able to provide access to our investigational drug outside of our clinical trials if certain conditions are met. Single Patient Expanded Access may not always be available as each patient is unique and this will be at the discretion of a physician based on many factors and conditions. To be eligible for access to the investigational medicine, a physician must apply and certify that the patient for whom the application is being submitted meets the following criteria:
Diagnosed with a serious, life-threatening or severely debilitating disease;
1. Unable to participate in SonALAsense clinical trials;
2. Have no comparable or satisfactory alternative treatment option available;
3. The risk/benefit profile of the investigational drug is favorable in light of the sufficient safety and efficacy information and the treating physician’s assessment of the patient’s medical condition.
Available Therapies via Single-Patient EA
Diffuse intrinsic pontine glioma
Recurrent or progressive glioblastoma
St. Jude Children's Research Hospital
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
Expanded access is used to describe treatment with an investigational product for a patient with a serious or life-threatening illness or condition when there are no comparable or satisfactory alternative treatments available and the patient is not eligible for a clinical trial. These investigational products are not approved by the U.S. Food and Drug Administration (FDA). The expanded access pathway is often used by commercial pharmaceutical companies to provide their products before they are approved.
St. Jude Children’s Research Hospital (St. Jude) is dedicated to its mission of advancing cures for pediatric catastrophic diseases through research and treatment. St. Jude provides the best possible care for our patients. We also make discoveries that will lead to better treatment for children and young adults around the world. We accomplish this by offering patients participation in clinical trials such as the LVXSCID-ND study (NCT01512888).
St. Jude Children’s Research Hospital is not a commercial pharmaceutical company and does not have the capability to manufacture products at an industrial level. Supplies of the investigational product for the LVXSCID-ND study are limited and earmarked for the completion of a prospective research guided clinical trial. At this time, the safety and efficacy of the product is under investigation. St. Jude is not able to provide the investigational product in the LVXSCID-ND study for expanded access. If your child has been diagnosed with X-Linked Severe Combined Immunodeficiency (SCID-X1), please contact the physician below to discuss possible treatment options.
Disease/Category-Specific EA Policies/Criteria
Stanford University- Center for Cancer Cell Therapy
Single-Patient EA Policies/Criteria
We currently do not accept requests for expanded access use of our investigational products. Supporting single patient expanded access would negatively impact our resources and ability to complete the active clinical investigations.
We encourage anyone interested in receiving investigational products manufactured for Stanford Center for Cell Therapy investigator-initiated trials to contact the Principal Investigator through ClinicalTrials.gov listing to see if they are eligible to enroll on an open study.
Steba Biotech S.A.
Available Therapies via Single-Patient EA
None are available via single-patient EA.
Disease/Category-Specific EA Policies/Criteria
Swedish Orphan Biovitrum AB
Single-Patient EA Policies/Criteria
Sobi may make an investigational medicine available if the following eligibility criteria and additional requirements are met:
§ The patient has a serious or life-threatening disease without a satisfactory alternative therapy or treatment and an unsolicited Managed Access request has been submitted to Sobi by the treating physician
§ The patient is not eligible or able to enter an ongoing clinical trial
§ There is sufficient data to indicate that the benefit of the investigational medicine outweighs the potential risks in the context of the disease or condition being treated
§ There is a sufficient supply of the investigational medicine available and providing the medicine will not interfere with ongoing clinical studies or other development activities
§ The patient meets any eligibility criteria set by Sobi’s Medical Committee evaluating Managed Access requests
§ Laws and regulations in the country of request allow Managed Access
Syncromune
Single-Patient EA Policies/Criteria
Syncromune is an oncology-focused biopharmaceutical company committed to developing therapies that may improve the lives of cancer patients. Our focus centers around conducting clinical research that evaluates the safety and effectiveness of new therapies for patients with solid tumor cancers. Our clinical trial programs are the primary way to get access to a Syncromune investigational therapy. These clinical trials provide the most effective way to assess how our investigational therapies may treat cancer, and are used to support regulatory approval.
At this time, Syncromune does not provide access to investigational therapies outside of clinical trials or prior to regulatory approval. You and your health care provider may learn more about our clinical trials by going to the clinical trials section of our website, or visiting ClinicalTrials.gov and searching for Syncromune.
If you are a health care provider who is interested in learning more about one of our investigational therapies, or a physician with questions about participation in one of our clinical trials, please submit a request to trials@syncromune.com. Syncromune will acknowledge your request as soon as possible, typically within 3 days of receipt.
If applicable, this website will be updated with hyperlinks to the relevant expanded access information on ClinicalTrials.gov. Syncromune reserves the right to revise this expanded access policy at any time.
Pursuant to the 21st Century Cures Act, the posting of policies by manufacturers and distributors shall not serve as a guarantee of access to any specific investigational therapy by any individual.
Available Therapies via Single-Patient EA
Metastatic castrate-resistant prostate cancer
Taiho Oncology, Inc
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
Taiho Oncology, Inc. (“Taiho”) is committed to helping patients with cancer obtain access to new treatments. While we believe this is best accomplished through participation in clinical trials, we recognize that this is not always possible for every cancer patient. In such situations, Taiho may be able to provide pre‑approval access to an investigational drug through our Expanded Access Program. Expanded Access, also known as Compassionate Use, is the use of an investigational medical product intended to diagnose, monitor or treat a patient's disease or condition, prior to FDA approval and outside of use in a clinical trial. Taiho accepts requests for pre‑approval access to investigational cancer medicines from physicians only.
To be eligible for access to an investigational medicine, a physician must apply and certify that the patient for whom the application is being submitted meets the following criteria*:
- The patient is suffering from a serious or life‑threatening malignancy and has received available standard treatments without success
- The patient is not eligible to participate in any ongoing clinical study of the Taiho investigational agent
- The medicine requested is part of an active ongoing clinical development program at Taiho Oncology (US) as described on our website: www.taihooncology.com/us under the tab for ‘Our Science’
- Provision of the investigational agent will not interfere with the ongoing Taiho development program, and the potential benefits to the patient outweigh the potential risks
A physician may click here to obtain an application form, submit the completed form, or submit questions about Taiho's Expanded Access Program, via email to ExpandedAccess@taihooncology.com. (For futibatinib specific requests, click here to obtain the application form).
* Note that meeting these criteria does not guarantee access to any investigational product.
Takeda Pharmaceuticals
Expected Application Timeframe
3 business daysDisease/Category-Specific EA Policies/Criteria
EXPANDED ACCESS TO INVESTIGATIONAL DRUGS
https://www.takedaoncology.com/medicines/expanded-access-to-investigational-drugs/
TenNor Therapeutics (Suzhou) Limited
Expected Application Timeframe
NAAvailable Therapies via Single-Patient EA
At this time, TenNor will not provide investigational drug outside of clinical studies and will reevaluate when sufficient preliminary safety and efficacy information has been obtained in clinical trials.
Rifasutenizol (formerly known as TNP-2198) is currently under clinical development for the treatment of Helicobacter pylori infection. Rifaquizinone (formerly known as TNP-2092) is currently under clinical development for the treatment of Prosthetic Joint infection (PJI) and Acute Bacterial Skin and Skin Structure Infections (ABSSSI).
At this time, TenNor will not provide investigational drug outside of clinical studies and will reevaluate when sufficient preliminary safety and efficacy information has been obtained in clinical trials.
Tetra Discovery Partners Inc
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
Tetra Therapeutics is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities.
Consistent with Tetra Therapeutic’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We understand that there are patients who will not be eligible for our clinical trials and may not have options for alternative therapies. In these circumstances, Tetra Therapeutics will consider providing a requesting physician with pre-approval access to a specific Tetra Therapeutics investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met and in accordance with the local regulatory process. These conditions include the following:
- The patient has a serious or life-threatening illness or condition;
- The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
- A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
- Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
- Adequate supply of the investigational drug is available.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.
Tetra Therapeutics is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Tetra Therapeutics may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Tetra Therapeutics, whose decisions are final.
Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to info@tetratherapeutics.com. All requests will be acknowledged within five business days if received during regular business hours and reviewed in a timely manner.
For further information on current clinical trials please see https://tetratherapeutics.com/clinical-trials/ and https://clinicaltrials.gov.
Available Therapies via Single-Patient EA
Zatolmilast
TFF Pharmaceuticals, Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Voriconazole Inhalation Powder for Pulmonary Aspergillosis and other voriconazole-sensitive pulmonary fungal infections
Available Therapies via Single-Patient EA
TFF Pharmaceuticals is developing its two lead drug candidates, Voriconazole Inhalation Powder and Tacrolimus Inhalation Powder, through the conduct of clinical trials. We understand that in some cases, patients may have exhausted other therapeutic options and/or may not qualify for participation in clinical trials. In such cases, treating physicians may request the use of an investigational drug on an expanded access or compassionate use basis. It is important to remember that the potential risks of an investigational drug are not yet established and therefore the treating physician must weigh the potential risks of an investigational drug against its potential benefits before considering the use of an investigational drug.
At this point, TFF Pharmaceuticals will consider providing a requesting physician with access to a specific TFF Pharmaceuticals investigational drug on an expanded access/compassionate use basis when certain conditions are met.
TFF Pharmaceuticals will consider requests for expanded access to Voriconazole Inhalation Powder:
• The patient is deemed by the patient’s physician to have a serious or life-threatening condition, with no satisfactory alternative therapies available, or has had an unfavorable response to adequate standard of care therapy.
• The patient’s physician believes that the use of Voriconazole Inhalation Powder is in the patient’s best interest.
• If provided, the investigational drug will be used in accordance with applicable laws and regulatory requirements of the country where the patient is treated.
• The patient is ineligible for or otherwise unable to participate in a clinical trial.
• Making the investigational drug available to a patient will not negatively impact or delay the conduct of ongoing clinical trials or regulatory review or approval of the investigational drug for broader patient access.
The treating physician must submit a formal request for expanded access to TFF Pharmaceuticals at expandedaccess@tffpharma.com. The treating physician is responsible for obtaining local regulatory and/or ethics/IRB approval for the investigational use. The request for expanded access must include at a minimum the requesting physician’s name and contact information, the indication, and rationale for request. Product specific eligibility criteria may apply and the treating physician will be requested to provide additional information.
The medical reviewers at TFF Pharmaceuticals will evaluate each request based on the criteria above, and the overall assessment of potential benefits and risks in the given patient.
TFF Pharmaceuticals may require more detailed information in order to fully evaluate a request. Every effort will be made to provide a response to an expanded access request within 3 business days of the formal request, with approval, non-approval or a request for additional information.
Additional information on Expanded Access for Voriconazole Inhalation Powder
Indications:
Invasive Pulmonary Aspergillosis
Chronic Pulmonary Aspergillosis
Allergic Bronchopulmonary Aspergillosis
Aspergillus Tracheobronchitis
Aspergillus bronchial anastomotic infection
Other voriconazole-sensitive pulmonary fungal infections
Eligibility Criteria:
Inclusion Criteria:
- Male or female aged 18 years or older at screening.
- Diagnosed with pulmonary aspergillosis including Invasive Pulmonary Aspergillosis (IPA), Chronic Pulmonary Aspergillosis (CPA), Allergic Bronchopulmonary Aspergillosis (ABPA), Aspergillus tracheobronchitis and Aspergillus bronchial anastomotic infection. Pulmonary infections with voriconazole sensitive fungi other than Aspergillus such as but not limited to scedosporium and fusarium are also allowed.
- Patient has limited or no treatment options due to documented or anticipated intolerance, toxicity, contraindications, or lack of clinical response to SOC antifungal therapy, as advocated by the relevant regional treatment guidelines.
- The treating clinician considers that the potential advantage of using Voriconazole Inhalation Powder outweighs the potential risks. In patients with disseminated fungal infection in addition to pulmonary infection, Voriconazole Inhalation Powder must be used as add-on therapy to the current SOC.
- The Sponsor agrees that the benefit:risk assessment is favorable for the use of Voriconazole Inhalation Powder in the patient.
- Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at Screening (Visit 1) and a negative urine pregnancy test (UPT) prior to first dose. Sexually active WOCBP and male patients must agree to use highly effective birth control or abstinence until 3 months after last dose.
- Patient succeeds in meeting training criteria on the use of the dry powder inhaler (DPI) and is willing and able to perform adequate inhalation technique for treatment duration in the opinion of the treating clinician or designee.
- Patient provides informed consent and agrees to follow the treatment regimen and safety and outcomes assessments.
Exclusion Criteria:
- Infection with fungi not responsive to voriconazole.
- Pregnant or breastfeeding.
- History or presence of hypersensitivity or idiosyncratic reaction to voriconazole or excipients in Voriconazole Inhalation Powder.
- Patients with severe liver disease as defined by Child-Pugh Class C.
- Patients who are eligible and are able to participate in a clinical trial of Voriconazole Inhalation Powder.
TG Therapeutics, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
TG Therapeutics is committed to bringing our investigational therapies for B-cell malignancies and autoimmune diseases to patients as fast as possible. We believe the best way for patients to access our investigational therapies is through participation in clinical trials, with the goal of obtaining regulatory approval of our products and allowing access to the broadest group of patients possible.
At this time, TG Therapeutics does not have a program to provide its investigational therapies to patients on an expanded access basis pursuant to section 561(b) of the U.S. Federal Food, Drug, and Cosmetic Act (FDCA). Because we do not currently have an expanded access program, we have not yet established procedures for making such requests or criteria for evaluating them. TG Therapeutics will update this policy if the company decides to make its investigational therapies available on an expanded access basis.
Patients may be able to access TG Therapeutics investigational therapies by participating in ongoing clinical trials. Whether or not a patient is eligible to enroll in any of our trials depends on a number of factors, including the specific requirements of each trial and the status of that trial. For more information about our trials, please visit Our Trials Overview or https://clinicaltrials.gov/ct2/results?cond=&term=%22tg+therapeutics%22&cntry=&state=&city=&dist.
TG is committed to responding to questions about its expanded access policy within five (5) business days of receipt. As noted above, at this time, we are not providing our investigational therapies on an expanded access basis.
Theriva Biologics
Expected Application Timeframe
5 DaysSingle-Patient EA Policies/Criteria
Purpose:
Theriva Biologics, is dedicated to developing innovative treatments for patients with cancer. We have developed this Expanded Access Policy to provide a pathway for patients who may benefit from treatment with our investigational product VCN-01 outside of clinical trials. As provided by the 21st Century Cures Act, we may revise this policy at any time.
Criteria for Expanded Access:
- Patient must have a confirmed diagnosis of a serious or life-threatening illness for which our investigational product is being investigated.
- Patient must have exhausted all standard treatment options and have no other viable treatment alternatives including participation in clinical trials.
- Patient must meet any additional criteria established by our Company to ensure the safe and appropriate use of our investigational product.
- There is sufficient evidence that the potential clinical benefit to the Patient of treatment with our investigational product outweighs the potential risks.
Process for Access:
- The Patient’s healthcare provider must submit a request for Expanded Access to our Company, including all relevant medical information and rationale for requesting access to our investigational product.
- Our medical team will review each request on a case-by-case basis to determine if the patient meets the criteria for expanded access.
- If approved, the Patient must provide written informed consent and our Company will work with the patient’s healthcare provider to facilitate access to our investigational product, including providing necessary information and support for administration of the drug.
- We will monitor the Patient’s response to treatment and report any adverse events to regulatory authorities as required.
- Expanded access does not guarantee a positive outcome and the patient must be informed of the potential risks and benefits of our investigational product.
Limitations:
- Expanded access may only be considered in circumstances where VCN-01 is available, and such expanded access will not compromise the scientific validity of broader development programs, or interfere with or delay current clinical trials or regulatory filings that are designed to make the therapy available to many more patients.
- Our company reserves the right to make final decisions on granting expanded access based on medical necessity and available supply of the drug.
Contact Information:
For more information on our Expanded Access Policy or to request access to our oncolytic adenovirus therapy, please contact our medical team at medicalmonitor@therivabio.com.
Approval:
This Expanded Access Policy has been approved by our Company’s executive team and will be reviewed periodically to ensure compliance with regulatory requirements and ethical standards.
Available Therapies via Single-Patient EA
VCN-01
Throne Biotechnologies Inc
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
Expanded Access to Investigational Drugs Policy:
1. Purpose and philosophy behind the policy
Throne Biotechnologies (Throne) is a clinical-stage therapeutic company with a disruptive stem cell technology that can fundamentally reverse type 1 diabetes (T1D), alopecia areata (AA) and other autoimmune diseases through immune education of Stem Cell Educator therapy (Gleukocellä) at the root causes. Over last 12 years, international multicenter clinical trials in the United States, China, and Spain have demonstrated the clinical safety and efficacy of Stem Cell Educator therapy. Throne is committed to bring this innovative therapy to help patients with serious and life-threatening diseases in medical need before it is fully approved by FDA for clinical use on prescription.
2. Approach to requests for access to Stem Cell Educator therapy
It is the first and most preferable route for patients to participate Throne’s ongoing clinical trials by using Stem Cell Educator therapy to treat type 1 diabetes and alopecia areata. If patients do not meet the criteria of clinical trials, their treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Throne's investigational products by contacting the Company.
3. Policy
3.1. Patient Eligibility Criteria: To be eligible for accessing to the investigational product, patients must meet following criteria:
• The patient has a serious or life-threatening condition with no satisfactory alternative.
• Assessment that benefits outweigh the risks to the patient.
• Adequate venous access for blood cell separation (apheresis).
• Ability to provide informed consent.
• Must agree to comply with all study requirements and be willing to complete all study visits.
3.2. Investigational Product Criteria:
• The product is under clinical investigation in one or more clinical trials, with high safety profiles.
• Assessment that the company has an adequate supply of the investigational product.
• A determination that expanded access will not interfere with the company's ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients.
3.3. Treating Physician Criteria and Responsibility: The physician must agree in writing to comply with:
• Physician(s) is (are) properly licensed.
• Physician(s) meet the applicable country-specific legal and regulatory requirements related to providing the investigational products under the Expanded Access.
• Any Throne’s requirements in terms of safety reporting and protection of intellectual properties.
• A treating physician may submit questions and requests regarding to the Expanded Access to email: connect@thronebio.com. We will do our best efforts to acknowledge each submitted request within 5 business days.
Tiziana Life Sciences LTD
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
Early (Expanded) Access Policy
Tiziana Life Sciences is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness.
Consistent with Tiziana Life Sciences’ mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing. At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Tiziana Life Sciences will consider providing a requesting physician with pre-approval access to a specific Tiziana Life Sciences investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:
The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
Adequate supply of the investigational drug is available.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.
Tiziana Life Sciences is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Tiziana Life Sciences may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Tiziana Life Sciences, whose decisions are final.
Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to ea@tizianalifesciences.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 5 business days after receipt.
TME Pharma
Single-Patient EA Policies/Criteria
At this time, TME Pharma is not offering Expanded Access for NOX-A12 (olaptesed pegol). Access to NOX-A12-based therapies is currently available only through participation in our clinical trials which allows us to allocate available supply to study the drug’s effectiveness and safety in a controlled and evaluable setting. For more information on our clinical trials that may be recruiting new patients, please consult www.clinicaltrials.gov. We understand the interest of patients in accessing NOX-A12 outside of clinical trials and prior to regulatory approval but we believe investigational products should be studied within the context of a clinical trial and encourage patients to enroll in a clinical trial wherever possible. We recognize the need for Expanded Access programs, and we will re-evaluate the status of our policy based on data from ongoing and future clinical trials.
Travere Therapeutics
Expected Application Timeframe
7 business daysTrellis Bioscience Inc.
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
The objective of our development program is to conduct clinical trials and to obtain marketing approval by the FDA and other regulatory authorities. Participation in clinical trials is the first and most preferable route. If participation in clinical trials is not an option, physicians may consider a single-patient expanded access IND. Requests are considered on an individual basis. The general criteria used to evaluate such requests for individual patients are as follows: 1) The patient is diagnosed with PJI; 2) Assessment that the company has an adequate supply of the investigational medicine; and 3) A determination that expanded access will not interfere with the company’s ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients. The company may request additional information, including patient history, in order to fully evaluate the request. If the physician will be the sponsor for the IND, he or she is required to obtain necessary ethical and regulatory clearances, including informed consent, as well as provide reporting on treatment outcomes.
Available Therapies via Single-Patient EA
Prosthetic joint infection.
Treovir LLC
Expected Application Timeframe
30 daysAvailable Therapies via Single-Patient EA
G207 for pediatric high grade gliomas
Trevi Therapeutics, Inc.
Expected Application Timeframe
5-7 business daysSingle-Patient EA Policies/Criteria
Trevi Therapeutics, Inc. is currently unable to offer Expanded Access.
Available Therapies via Single-Patient EA
Currently, Trevi Therapeutics, Inc. does not have any investigational products available for Expanded Access.
Disease/Category-Specific EA Policies/Criteria
Trinomab Biotech Co., Ltd
Single-Patient EA Policies/Criteria
Trinomab is dedicated to discovering and developing novel native human monoclonal antibody (mAb) drug. Trinomab’s lead product, TNM002 injection, is the first recombinant native human mAb against tetanus toxin and is indicated for prophylaxis against tetanus. Currently, TNM002 has completed phase I clinical trials. A phase II clinical trial is ongoing. The results of clinical trials show that TNM002 has excellent safety and efficacy profiles, with low risk of immunogenicity. At Trinomab, we focus on conducting clinical trials aimed at gaining regulatory approval, making TNM002 available to patients as early as possible. Trinomab understands the interest of patients in accessing TNM002 outside of clinical trials and prior to regulatory approval for potentially life threatening diseases, however we do not currently offer any Expanded Access Programs for TNM002. Trinomab recognizes the need for Expanded Access Programs and will continue to evaluate of our policy based on data from ongoing and future clinical trials and the clinical development plan in the U.S.
Trinomab Biotech Co., Ltd
Single-Patient EA Policies/Criteria
Trinomab is dedicated to discovering and developing novel native human monoclonal antibody (mAb) drug. Trinomab’s lead product, TNM002 injection, is the first recombinant native human mAb against tetanus toxin and is indicated for prophylaxis against tetanus. Currently, TNM002 has completed phase I clinical trials. A phase II clinical trial is ongoing. The results of clinical trials show that TNM002 has excellent safety and efficacy profiles, with low risk of immunogenicity. At Trinomab, we focus on conducting clinical trials aimed at gaining regulatory approval, making TNM002 available to patients as early as possible. Trinomab understands the interest of patients in accessing TNM002 outside of clinical trials and prior to regulatory approval for potentially life threatening diseases, however we do not currently offer any Expanded Access Programs for TNM002. Trinomab recognizes the need for Expanded Access Programs and will continue to evaluate of our policy based on data from ongoing and future clinical trials and the clinical development plan in the U.S.
Triumvira Immunologics, Inc.
Expected Application Timeframe
3 business daysSingle-Patient EA Policies/Criteria
Triumvira Immunologics, Inc. is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness. Consistent with Triumvira, Inc.’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing.
At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Triumvira, Inc. will consider providing a requesting physician with pre-approval access to a specific investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:
• The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.
Triumvira is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Triumvira may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Triumvira whose decisions are final. Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to Partners@Triumvira.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt.
Available Therapies via Single-Patient EA
TAC01-CD19, autologous TAC (T cell antigen coupler) T cells, single infusion, multiple dosage levels.
Indication: B-Cell Lymphoma
TrueBinding Inc.
Expected Application Timeframe
7 daysSingle-Patient EA Policies/Criteria
The FDA has developed a guidance whereby patients who have no treatment alternatives and cannot participate in a clinical trial may receive investigational treatments from companies. This program is called Expanded Access. In order to be eligible, patients must demonstrate that they have exhausted all available medications to treat their disorder, meaning they have taken available medications and have no further benefit, are intolerant to other medications, or have contraindications to other medications. They must also demonstrate that there are either no clinical trials available in their locale, or that they are not eligible to participate in other clinical trials. The key decision for physicians and patients is whether they believe the benefits of treatment outweigh the risks.
TrueBinding has prepared an expanded access protocol for which treating physicians can apply on behalf of their patients. Once enrolled in the protocol, patients receive monthly (every 28 days) IV infusions of TB006. Treatment may continue indefinitely, however it may be discontinued whenever desired. The protocol contains some patient monitoring suggestions, however other than adverse events there are no data to report to TrueBinding.
You may receive a copy of the protocol and the Investigator’s Brochure by contacting us. A Confidentiality Disclosure Agreement will be sent to you. Once executed, the documents will be sent. If you have a patient who qualifies and is interested, you may apply for the program by accessing the application. You will be notified of approval within 7 days of the application. If the application is approved, you must participate in a short training session. This covers preparation of the IV drug product, collection and reporting of adverse events, and other elements of Good Clinical Practices (GCPs).
When this is complete, drug will be sent to you. You must provide IV administration supplies, including saline, tubing, and administration equipment.
You may receive a copy of the protocol and the Investigator’s Brochure by contacting us. A Confidentiality Disclosure Agreement will be sent to you. Once executed, the documents will be sent. If you have a patient who qualifies and is interested, you may apply for the program by accessing the application. You will be notified of approval within 7 days of the application. If the application is approved, you must participate in a short training session. This covers preparation of the IV drug product, collection and reporting of adverse events, and other elements of Good Clinical Practices (GCPs).
When this is complete, drug will be sent to you. You must provide IV administration supplies, including saline, tubing, and administration equipment.
Available Therapies via Single-Patient EA
TrueBinding, Inc. is pleased to offer their lead pipeline compound, TB006, on a compassionate use basis to patients with Alzheimer’s disease (AD) and related dementias. TB006 is a humanized monoclonal antibody which targets galectin-3 (Gal-3), an endogenous protein involved in a number of neurological and metabolic disorders. There are two antibodies approved for AD, aducanumab (AduhelmÒ) and lecanemab (LeqembiÒ). TB006 differs from these antibodies in two important ways. First, the two approved antibodies are direct inhibitors of beta amyloid, the protein involved in the formation of neurofibrillary tangles and amyloid plaques in the brain. TB006, by blocking the action of Gal-3, inhibits not only beta amyloid but precursors of other oligomers and neurofibrillary tangles such as tau, alpha-synuclein, and apolipoprotein E4. Secondly, the two approved antibodies are indicated in AD patients who are in their early phase (MCI-early AD) and have PET imaging evidence of amyloid plaques. TB006 is being studied in patients with established disease on the mild-moderate-severe (Mini Mental Status Exam score range of 24 or less) regardless of their amyloid status.
TVAX Biomedical
Expected Application Timeframe
48 hoursSingle-Patient EA Policies/Criteria
TVAX Biomedical, Inc. (TVAX) is committed to developing immunotherapies for patients with serious cancers. Our goal is to provide access to our investigational therapies primarily through clinical trials. At this time, TVAX does not have any investigational immunotherapy product available for expanded access.
“Expanded Access” refers to the use of an investigational therapy outside of a clinical trial for potential treatment of a serious condition in a patient. The US Food and Drug Administration has set forth guidelines when considering expanded access.
TVAX is not currently making its investigational immunotherapy product candidates available on an expanded access basis anywhere in the world. Requests for expanded access to TVAX Biomedical’s investigational immunotherapy product candidates must come from the patient’s treating physician because only physicians can enroll patients in a clinical trial or the expanded access program.
Certain therapies, like TVAX Immunotherapy, are made through complex manufacturing processes. TVAX seeks to retain the ability to make and supply product in a fair and equitable manner and in a volume that assures adequate manufacturing capacity for clinical trials and development programs. TVAX believes that participation in one of its’ clinical trials is the most appropriate way to access these investigational therapies.
At this time, TVAX is not currently making its unapproved therapies available on an expanded access or right to try basis. In the event TVAX decides to consider expanded access or right to try use, TVAX will evaluate and respond to each request that it receives on a case-by-case basis.
If you have questions, please speak with your physician or contact TVAX at info@tvaxbiomedical.com.
Consistent with the 21st Century Cures Act, TVAX may revise this policy at any time.
UCB
Expected Application Timeframe
N/AUltimovacs ASA
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Ultimovacs is currently unable to offer an Expanded Access Program to patients
Available Therapies via Single-Patient EA
There are no investigational products available for Expanded Access
Disease/Category-Specific EA Policies/Criteria
Ultragenyx
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
Requests for early access to investigational therapies must be made by a qualified physician.
UNION therapeutics
Expected Application Timeframe
2-4 business daysSingle-Patient EA Policies/Criteria
At this point of clinical development, UNION has not established an Expanded Access Program that allows patients access to orismilast outside of clinical trials. Participation in clinical trials will ensure dedicated monitoring of the efficacy and safety of the drug, which is currently the most appropriate and responsible setting of patient treatment.
Disease/Category-Specific EA Policies/Criteria
Usona Institute
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Licensed physicians should submit expanded access requests to Usona at EA@usonainstitute.org using the Usona Expanded Access Request Form. Requests for Expanded Access will be acknowledged within 5 business days of receipt. All Expanded Access use requests will be decided on a case-by-case basis at the sole discretion of Usona. All requests received will be reviewed anonymously by an internal Usona Committee to ensure patient safety and equality. The licensed treating physician must obtain, where applicable, all Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals along with approval from the overseeing regulatory authority (e.g. FDA, EMA, Health Canada, etc.) prior to submission.
Expanded Access requests to Usona must meet ALL of the following criteria in order to be considered:
• The disease or condition for which use is requested is serious or life-threatening;
• The patient is ineligible or not able to participate in a clinical trial for the requested use and all approved treatment options have been exhausted without success and no satisfactory alternative treatment is available as determined by the requesting licensed physician;
• The requesting physician is a licensed physician and is authorized to deliver treatment as outlined in the request;
• There is sufficient clinical evidence to inform the safe use of the investigational drug under the requested use (at the requested dose and frequency of treatment);
• There is sufficient clinical evidence to suggest the requested use is expected to provide a potential clinical benefit to the patient (at the requested dose and frequency of treatment);
• The requested use would not negatively impact or interfere with active clinical trials or drug development programs of the applicable investigational drug;
• The treating physician has received approval by their overseeing regulatory authority (e.g. FDA, EMA, etc.) and where applicable, any Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals
• The requested use complies with all applicable laws, rules and regulations;
• The requested use complies with Usona’s policies, including strict adherence to the ‘Usona Expanded Access Set and Setting Manual’;
• There is an adequate supply of the requested investigational drug available for the requested use.
Please note that Usona is only able to respond to Expanded Access requests from licensed physicians. Additional requirements apply to Expanded Access use of controlled substances, including specific personnel and facility requirements for expanded access use of drugs of the psychedelic drug class.
Available Therapies via Single-Patient EA
Psilocybin capsules (25 mg)
Disease/Category-Specific EA Policies/Criteria
USWM, LLC dba US WorldMeds
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
US WorldMeds is committed to serving patients by providing safe, fair, and sustainable patient access to its medicines. USWM may be able to provide patients access to its investigational drug, DFMO (also called eflornithine or difluoromethylornithine), outside of a clinical trial if certain conditions are met and if permitted by local law and regulation. The use of an investigational medical product (i.e., one that has not been approved by the Food and Drug Administration [FDA]) outside of a clinical trial is known as Expanded Access (sometimes referred to as “compassionate use”). Generally, there are two types of Expanded Access—Group Expanded Access and Individual Patient Expanded Access. For more information on the different types of Expanded Access, visit the following FDA website: https://www.fda.gov/news-events/public-health-focus/expanded-access.
USWM collaborates with Beat Childhood Cancer Research Consortium (BCC) and Sponsor-Investigator, Dr. Giselle Sholler and makes investigational product DFMO 192 mg tablets available for pediatric oncology indications through multiple clinical trials and a Group Expanded Access Program (also called Intermediate Population Expanded Access).
USWM does not currently provide Individual Patient Expanded Access; however, detailed written requests for unique circumstances will be reviewed on a fair and equitable basis.
Expanded Access may not always be available. Information about the Group Expanded Access Program, including a full list of eligibility criteria and site contact information can be found here: https://clinicaltrials.gov/ct2/show/NCT03581240. In addition to meeting the eligibility criteria outlined at the link above, the following criteria must be met for entry into the Group Expanded Access Program:
- USWM must have sufficient supply of DFMO to reasonably accommodate the anticipated duration of treatment.
- The patient’s licensed physician must be willing to collaborate with the enrolling center.
- The patient’s physician and enrolling physician determine there is no comparable or satisfactory therapy available to treat the patient’s disease or condition and they agree that the patient is clinically stable and able to receive this medication.
- The patient’s parent/guardian is willing to travel with the patient to BCC at study entry and every three months while on treatment.
Licensed physicians may contact USWM with general requests or questions regarding Expanded Access at regulatoryaffairs@usworldmeds.com. USWM will endeavor to acknowledge general requests and questions within 5 business days. In line with the 21st Century Cures Act, USWM cannot guarantee access to investigational product to all patients. USWM may revise this policy at any time; at such time, the revised policy will be made publicly available.
Vasgene Therapeutics, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
We ask that the physician in charge of the patient ask for Pre-approval access to investigational drug. Each request will be reviewed by a physician to assure that the eligible patients can receive pre-approval investigational drug.
Decisions for Pre-Approval Access to the investigational medicine will be based on the safe use in the patient, given the investigational nature of these medicines that have not completed clinical trials for the specific disease and state. Next is the likelihood of benefit for the patient based on the clinical results of the investigational medicine. Discussion with the physician managing the patient will help determine if the investigational agent is the best option for the patient. Decisions for drug access will be made in a fair and equitable process. There is no guarantee that each request will be fulfilled.
We must also ensure that by providing the drug outside the clinical trial the completion of the trial, and thus drug development plan, regulatory review and approval is not adversely affected.
Request for Pre-Approval Access must be made by your physician who will request the drug from VasGene Therapeutics, Inc. VasGene will respond to the request within 5 business days.
Below are the Eligibility Criteria for the request:
· The illness must be serious or life threatening.
· There are no other viable options (including approved products or active clinical trials).
· There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks based on
what is known at present.
· There is a plan in place to make the medicine, once approved, available commercially.
· Whether or not sufficient quantities of the investigational medicine are available.
· Additional regarding the therapy, its development program, the patient, or other circumstances may impact eligibility.
Available Therapies via Single-Patient EA
sEphB4-HSA
Vaxxinity, Inc.
Single-Patient EA Policies/Criteria
Vaxxinity is committed to developing safe and effective immunotherapeutic vaccines, with a goal of providing our next generation products as soon as possible through efficient product development. At this time, Vaxxinity’s investigational products can only be accessed through participation in a clinical trial. Vaxxinity does not currently offer an expanded access program.
Available Therapies via Single-Patient EA
Currently, Vaxxinity has no products available via single-patient EA.
Vedanta Biosciences, Inc.
Single-Patient EA Policies/Criteria
At present, Vedanta has not established an expanded access program and does not offer expanded access to our investigational product candidates outside of a clinical trial.
Disease/Category-Specific EA Policies/Criteria
VelosBio Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
VelosBio will review all requests for investigational drug access on a case-by-case basis, taking into consideration regulations and guidance issued by applicable health authorities. Among other criteria, VelosBio will consider whether:
- The patient has a serious, debilitating, or life-threatening medical condition for which no other medically appropriate treatment is available;
- The patient’s healthcare provider can provide an assessment of the benefits and risks of the proposed expanded access use that suggests that the potential benefits of access to the investigational therapy likely outweigh known potential risks in the context of the risks posed by the patient’s underlying medical conditions;
- The patient’s healthcare provider can provide relevant data (eg, medical records, etc.) to support the assessment of benefits and risks, considering standards of patient confidentiality appropriate for the situation;
- The patient’s healthcare provider is currently licensed with the appropriate practitioner licensing board;
- The patient’s healthcare provider is willing and able to support any regulatory procedures required by the relevant regulatory authority;
- The patient’s healthcare provider is willing to enter into an expanded access agreement with VelosBio;
- The patient’s healthcare provider will obtain informed consent from the patient or his/her legal guardian(s) for use of the investigational therapy;
- The patient’s healthcare provider will supervise administration of the investigational therapy in accordance with any procedures specified by VelosBio (which in some countries may include a protocol for use of the investigational therapy);
- The patient’s healthcare provider is willing and able to report any side effects of the investigational therapy and any other results as requested by VelosBio to assess benefits and risks of the expanded access use;
- The patient does not qualify to participate in any ongoing clinical trial sponsored by VelosBio in an accessible geographical location;
- Sufficient data exist to support the proposed expanded access use and the assessment of the benefits and risks of the expanded access use;
- Access would not impede or compromise the ongoing clinical development program or regulatory approval of the investigational therapy; and
- There is adequate supply of the investigational therapy to support access and such supply will not negatively affect a clinical trial or anticipated product launch
Requests for access to a VelosBio investigational therapy must be made by the patient’s treating physician. Whenever possible, the patient, the patient’s parent/legal guardian, or patient caregiver should work through the treating physician rather than contacting VelosBio directly.
To initiate a request for access to a VelosBio investigational drug, the physician should email info@velosbio.com. Such requests should aim to address Criteria 1-10 above. VelosBio will strive to respond to requests for access of an investigational product within five (5) business days of receiving a complete request that includes all necessary documentation.
Venatorx Pharmaceuticals, Inc.
Expected Application Timeframe
Up to 24 hoursSingle-Patient EA Policies/Criteria
Venatorx Pharmaceuticals (the “Company”) is focused on conducting clinical studies and obtaining regulatory approval of investigational medicines in support of its mission to develop safe and effective treatments for infectious diseases. The best way for patients to get access to investigational medicines is by taking part in clinical trials. Please search ClinicalTrials.gov for publicly available information related to Venatorx’s ongoing clinical trials.
It is recognized that not all patients are eligible to enroll in clinical trials. However, at this time, Venatorx is not able to offer Expanded Access (“EA”) and will not accept EA requests.
Venatorx recognizes the need for EA programs. The Company is currently establishing the necessary processes and procedures, as well as putting in place resources and supplies so that an EA program may be established in the future.
If you have questions about Venatorx’s EA policy, please contact medinfo@venatorx.com.
Available Therapies via Single-Patient EA
Cefepime/VNRX-5133 (taniborbactam)
Veralox Therapeutics Inc
Expected Application Timeframe
48 hoursSingle-Patient EA Policies/Criteria
At this time, we are not yet providing Expanded Access.
Once our clinical trials have progressed to Phase 3 and we are able to provide Expanded Access, to be eligible for access to one of our investigational therapies via an Expanded Access mechanism, a physician must certify that the patient for whom the request is being submitted meets the following criteria*:
• The patient has received all available standard treatments without success
• The patient is not eligible to participate in any ongoing clinical study of a suitable investigational therapy
• The investigational therapy requested is part of an active ongoing clinical development program as described on our website
• Provision of the investigational therapy will not interfere with the ongoing development program, and the potential benefits to the patient outweigh the potential risks
* Note that meeting these criteria does not guarantee access to any investigational product.
Receipt of a request will be confirmed electronically within 48 hours. Following receipt of the request, we may require additional information as needed to complete our assessment. Once all of the necessary information is provided for review by our medical experts, a decision for expanded access will be communicated to the Physician within 10-15 business days.
Available Therapies via Single-Patient EA
At this time, we are not yet providing Expanded Access.
Viela Bio
Expected Application Timeframe
24 hoursSingle-Patient EA Policies/Criteria
At Viela Bio, we work every day to improve the lives of people impacted by serious, underserved, inflammatory and autoimmune diseases. We share the urgency of patients seeking new treatments for potentially life-altering diseases, and understand the interest in accessing our therapies outside of clinical trials and prior to regulatory approval.
Expanded access, also referred to as compassionate use, is a channel through which the US Food and Drug Administration (FDA) allows physicians to request investigational medicines for patients. We do not currently offer any expanded access programs, as we are focusing our resources – including supply of complex investigational medicines – on clinical trials and regulatory approval.
We encourage patients who are interested in accessing therapies in our pipeline to talk to their doctor about participating in a clinical trial. Information about all of our trials, including eligibility criteria and locations, is available at ClinicalTrials.gov.
If we are able to offer expanded access in the future, we will update this policy. If you have further questions, please email eap@vielabio.com.
Vivoryon Therapeutics NV
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Expanded Access
Under certain circumstances, a person suffering a serious or life-threatening disease may ask to use an experimental treatment outside a clinical trial, before its safety and efficacy have been fully evaluated, and before the regulatory authorities have approved it. Generally, this is an option only for patients who have exhausted all available medical options and do not qualify for the ongoing clinical trials.
Vivoryon understands the intent of expanded access programs but, at this time, we can best advance the development of these potential promising products by enrolling patients in clinical trials which are designed to enable approval allowing access for the broader patient community. Our priority is to demonstrate the safety and effectiveness of varoglutamstat (PQ912), our investigational oral small molecule medicine in development to treat Alzheimer’s disease, in order to obtain regulatory approval and make it available to appropriate patients as rapidly as possible. We do this by running a thoughtfully designed and robust clinical trial program.
We are currently unable to offer expanded access for varoglutamstat (PQ912) and we believe that participation in one of our clinical trials is the most appropriate way to access our investigational therapies.
More Information
Treating physicians, patients and/or caregivers interested in learning more about Vivoryon’s investigational oral small molecule medicines currently undergoing clinical studies can find more information here or by or visiting www.clinicaltrials.gov and searching for Vivoryon Therapeutics.
If you are a health care provider who is interested in learning more about one of our investigational therapies, or a physician with questions about participation in one of our clinical trials, please submit a request to clinics@vivoryon.com. Vivoryon will acknowledge questions as soon as possible, usually within 5 business days of receipt.
If applicable, this website will be updated with hyperlinks to the relevant expanded access information on www.clinicaltrials.gov upon activation. As authorized by and in accordance with the 21st Century Cures Act, Vivoryon reserves the right to revise this policy at any time.
vTv Therapeutics Inc.
Expected Application Timeframe
N/ASingle-Patient EA Policies/Criteria
At this time, vTv Therapeutics does not offer expanded access to any of our investigational drugs.
WEX Pharmaceuticals Inc.
Expected Application Timeframe
5 Business DaysSingle-Patient EA Policies/Criteria
An Early Access program for Halneuron may be opened if WEX determines that all of the following criteria are met:
• The disease or condition is seriously debilitating or serious.
• The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
• The patient is not eligible to participate in a suitable clinical trial
• An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
• There is adequate available data to permit selection of an appropriate dose level
• Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
• There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient
• The patient, treating physician, regulatory authority and the company agree to the use of Halneuron
• Supplied drug is used as clinically indicated from the product information supplied by Halneuron and in discussion with the treating physician
DISCLAIMER: An existing program does not guarantee availability in all countries. Various regulatory mechanisms exist in different countries to provide Early Access to new medicines and as a result, country specific variations for Early Access will occur. Any Early Access to medicines must always comply with the applicable country-specific laws and regulations including medicines importation requirements.
Available Therapies via Single-Patient EA
1. Moderate-to-severe chemotherapy-induced neuropathic pain
2. Post-burn pain
3. Post-burn itch
4. Chronic pain
xCures Inc.
Expected Application Timeframe
2 business daysDisease/Category-Specific EA Policies/Criteria
Xynomic Pharmaceuticals, Inc.
Expected Application Timeframe
5 business daysSingle-Patient EA Policies/Criteria
Xynomic Pharmaceuticas, Inc. (Xynomic) is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness.
Xynomic development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational products, and to obtain regulatory approval.
Xynomic is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Xynomic investigational products by contacting the Company.
Any use of a Xynomic investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including Xynomic policies and procedures.
Available Therapies via Single-Patient EA
Abexinostat tablets for treatment of advanced renal cell carcinoma in combination with pazopanib. Xynomic will only provide abexinostat tablets.
Zhejiang DTRM Biopharma Co. Ltd.
Single-Patient EA Policies/Criteria
Zhejiang DTRM Biopharma Co. Ltd. is an oncology-focused Biopharmaceutical company committed to delivering medicines that provide a better life for cancer patients. Our development resources are focused on conducting scientific research that evaluates the safety and effectiveness of new medicines for patients with cancer. Our clinical trial programs are the primary way to get access to one of Zhejiang DTRM Biopharma’s investigational medicines. These clinical trials provide the most effective way to assess how our investigational medicines may treat cancer and are used to support regulatory approval. Zhejiang DTRM Biopharma encourages patients to speak with their treating physicians about participating in a clinical trial, when possible. You can find additional information about Zhejiang DTRM Biopharma’s ongoing clinical trials by accessing https://clinicaltrials.gov.
Where enrollment into a clinical trial is not an option, and where all currently available treatment options have been exhausted, an investigational medicine may be provided prior to regulatory approval or commercial availability, under a provision referred to as “Expanded Access” (also commonly called “Compassionate Use”) by the U.S. Food and Drug Administration (“FDA”).
Expanded Access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational drug for treatment outside of a clinical trial when no comparable or satisfactory alternative therapy exists. Per FDA, Expanded Access may be appropriate when all of the following apply:
1. A patient has a serious disease or condition, or a patient’s life is immediately threatened by their disease or condition.
2. There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
3. Patient enrollment in a clinical trial is not possible.
4. Potential patient benefits justify the potential risks of treatment.
5. Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
As a general policy, Zhejiang DTRM Biopharma will not provide investigational medicines to patients until a dose and schedule has been well established for the agent, preliminary data exists that the agent has some evidence of activity in a particular indication, and the agent is found to be safe as a result of a risk-benefit evaluation.
Zhejiang DTRM Biopharma is not currently making any of its investigational medicines available on an Expanded Access basis prior to regulatory approval anywhere in the world. Zhejiang DTRM Biopharma may reconsider making one or more of its investigational medicines available through an Expanded Access Program in the future. As authorized by the 21st Century Cures Act, Zhejiang DTRM Biopharma may revise this Expanded Access Policy at any time.
If you have additional questions, please speak with your treating physician or contact Zhejiang DTRM Biopharma at dtrm-555fdctablets@dtrmbiopharma.com