Company Directory

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

Currently, ASC Therapeutics does not offer an expanded access program. We are early in the development and are still evaluating the risks associated with its investigational gene therapy, mainly related to immune-related side effects at administration, assessment of the optimal dose, understanding of the duration of treatment response, and long-term loss of some or all treatment effects. For these reasons, ASC Therapeutics has decided not to provide expanded access to its investigational gene therapy until there is clarity on these issues, a decision made after thorough consideration and in consultation with our hemophilia advisory board. Currently, access to our investigational therapy is possible only through participation in clinical trials.

Disease/Category-Specific EA Policies/Criteria

https://www.asctherapeutics.com/expanded-access-policy/

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