Company Directory

Single-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.

Available 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.

Single-Patient EA Policies/Criteria

Currently, Adagio does not have an expanded access program that permits access to our investigational products prior to regulatory authority (such as FDA) authorization.

Single-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).

Single-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.

Disease/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.

Single-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)

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.

Single-Patient EA Policies/Criteria

http://www.alkermes.com/assets/content/files/Expanded%20Access%20Policy.pdf

Single-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.

Disease/Category-Specific EA Policies/Criteria

http://www.alnylam.com/medical-professionals/expanded-access-program/

Single-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

Single-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

Single-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.

Single-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

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.

Single-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/

Single-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

https://aravive.com/patients/

Single-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

http://arcellx.com/#pipeline

Single-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

Single-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.

Single-Patient EA Policies/Criteria

Disease/Category-Specific EA Policies/Criteria

Single-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.

Single-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

Single-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

http://www.atoxbio.com/pipeline/aki/overview/

Single-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

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.

Single-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 

Single-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

https://belitebio.com/#product-008

Single-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.

Single-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).

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.

Single-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

Single-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

Single-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)

Single-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.

Single-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.

Single-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.

Available Therapies via Single-Patient EA

Intermediate-Size Patient Population Expanded Access Program for Intravenous Difelikefalin

The purpose of this protocol is to provide access to IV difelikefalin to a limited number of patients undergoing hemodialysis with moderate-to-severe pruritus during the period prior to commercial availability.

Available Therapies via Single-Patient EA

None

Disease/Category-Specific EA Policies/Criteria

https://celldex.com/docs/Compassionate_Use_Policy_MAY2018.pdf

Single-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.

Single-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.

Single-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

Single-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

Single-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

Single-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.

Single-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

https://www.daiichisankyo.com/rd/pipeline/index.html

Single-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)

Single-Patient EA Policies/Criteria

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, DZD9008, is an oral EGFR tyrosine kinase inhibitor (TKI). It is currently being investigated as a single agent in a phase 2 clinical trial 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 DZD9008 available to all patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access DZD9008 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 DZD9008, 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 DZD9008.

Dizal 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.

Single-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

Single-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

https://erytech.com/contact/compassionate-use/

Single-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.

Single-Patient EA Policies/Criteria

Exelixis believes that the best way for a patient to have access to an investigational medication is via 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 www.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. 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 to Exelixis.

Single-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

https://www.fortebiorx.com/pipeline/default.aspx

Single-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.

Single-Patient EA Policies/Criteria

Currently, FUJIFILM Pharmaceuticals U.S.A., Inc. is not accepting expanded access/compassionate use requests.

Single-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.

Single-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

http://www.gannexpharma.com

Single-Patient EA Policies/Criteria

https://www.gene.com/patients/investigational-medicines/criteria

Single-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

https://www.gensight-biologics.com/wp-content/uploads/2018/03/211012_GenSight-Biologics_Expanded-Access-Progam-Policy_GS030.pdf

Single-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.

Single-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

Single-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:

FOCUS Phase I/II Trial

EXPLORE Phase II Trial

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

https://www.gyroscopetx.com/expanded-access/

Single-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

Single-Patient EA Policies/Criteria

HiberCell, Inc. is committed to developing novel therapeutics that address the most common cause of cancer mortality: relapse and metastasis. HiberCell has multiple compounds in development focused on modulating the immunosuppressive tumor microenvironment and adaptive stress biology. Currently HiberCell has two therapies in clinical testing. One of its therapies, Imprime PGG, an innate immune modulator, has advanced into clinical testing (Phase 2). Clinical studies evaluating the safety and efficacy of Imprime PGG in combination with immune checkpoint inhibitors, tumor-targeting, and anti-angiogenic antibodies for the treatment of multiple cancers have been conducted. HiberCell’s second therapy,  HC-5404, an ER stress modulator PERKi treatment, has entered into clinical testing (Phase 1) and the first clinical study evaluating the dose, safety and efficacy of HC-5404 is underway. At this time, HiberCell does not have a compassionate use program; however, it will consider providing Imprime PGG or HC-5404 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, HiberCell 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 physician or a potential patient and your physician believes you may be a candidate for single patient access to Imprime PGG, please ask your doctor to contact Michele Gargano, VP Clinical Operations, HiberCell, at mgargano@hibercell.com, who will respond within 5 working days.

Available Therapies via Single-Patient EA

Imprime PGG, for multiple oncology indications, in combination with checkpoint inhibitor, tumor-targeting- or anti-angiogenic antibodies

HC-5404 for the treatment of renal cell carcinoma and other solid tumors including gastric cancer, metastatic breast, and small-cell lung carcinoma, or other select tumors.

Disease/Category-Specific EA Policies/Criteria

https://www.hibercell.com/programs/

Single-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

https://iliadbio.com/clinical.html

Single-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

Single-Patient EA Policies/Criteria

http://immixbio.com/pipeline/compassionate-use-policy/

Available Therapies via Single-Patient EA

imx-110 - all advanced solid tumors

Single-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

http://immuneonco.com/info.php?class_id=103102

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. 

Single-Patient EA Policies/Criteria

Immunicum is currently unable to offer an Expanded Access

Available Therapies via Single-Patient EA

Currently, Immunicum does not have any investigational cell therapy products
available for Expanded Access

Single-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.

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.

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

Single-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.

Single-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.

Single-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)

Single-Patient EA Policies/Criteria

http://www.janssen.com/sites/www_janssen_com/files/janssen_pre-approval_access_policy_022017.pdf

Single-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.

Single-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.

Available Therapies via Single-Patient EA

Paxalisib (formerly known as GDC-0084)
 

Disease/Category-Specific EA Policies/Criteria

https://www.kaziatherapeutics.com/researchpipeline/paxalisib

Single-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.

Single-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

Single-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.

Single-Patient EA Policies/Criteria

Poteligeo was approved by the FDA on August 8, 2018 for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. Only patients who have relapsed or refractory MF or SS after at least one prior systemic therapy will be eligible to participate.
The main purpose of the EA program is to facilitate the access of Poteligeo to patients who lack therapeutic alternatives and in which there is a reasonable expectation that the provision of access to Poteligeo will have a positive benefit/risk for the patient. The intention was for the EA program to fill the time between approval by the FDA and the date of availability of commercial supply to patients. Commercial supply is now available and the EA program ended on November 1, 2018.

Disease/Category-Specific EA Policies/Criteria

https://www.leaptx.com/for-patients

Single-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

Single-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

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

Single-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.

Single-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.

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.

Single-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. 

Single-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.

Single-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

https://nrg1.com/

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.

Single-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

http://metvital.com/

Available Therapies via Single-Patient EA

Currently, none.

Single-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.

Single-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

Single-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

Single-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).

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.

Single-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.

Single-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)

Single-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

https://www.myeloidtx.com/products

Single-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/

Single-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

Single-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.

Available Therapies via Single-Patient EA

Novartis considers granting managed access to investigational or pre-approval products via single patient access provided the Managed Access criteria are fulfilled.

Single-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

Single-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.

Single-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)

Single-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.

Single-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

Single-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.

Single-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

Single-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.

Single-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

Early Access Policy