Company Directory

Single-Patient EA Policies/Criteria

Abbisko aspires to discover and develop differentiated therapeutics for the treatment of cancer diseases. One of the Abbisko’s lead products, ABSK021, is an oral, potent, highly selective CSF-1R inhibitor. It is currently being investigated as a single agent in one Phase 3 clinical trial for the treatment of patients with TGCT (Tenosynovial Giant Cell Tumor). We are conducting clinical trials aiming at obtaining regulatory approval to make ABSK021 available to all patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access ABSK021 at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at clinicaltrials.gov..

Abbisko understands the interest of patients in accessing ABSK021 outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases, however we do not currently offer any Expanded Access Programs for ABSK021.

Abbisko recognizes the need for Expanded Access Programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.

Single-Patient EA Policies/Criteria

This is the physician-sponsored, single-patient extend access (EA) to ABM-1310. ABM-1310 is an investigational small molecule inhibitor for treating patients with BRAFv600 mutant malignancies. This program currently is only available through licensed physicians in the United States and is offered to those patients who previously participated in an ABM-1310 clinical trial in the U.S., to allow them to continue receiving ABM-1310 therapy. ABM Therapeutic (ABM) is the ABM-1310 drug provider and may provide possible assistance to the physicians for their EA applications.
Physician applicants are responsible for submitting required documents and obtaining all required approvals prior to requesting ABM-1310 drug supply. ABM reserves the right to review, approve or reject EA applications based on case-by-case evaluation. The ABM-1310 drug supply is free of charge to treating physicians and patients after all approvals are granted. All patient care is the responsibility of the treating physician and the patient’s own healthcare coverages.

 

Available Therapies via Single-Patient EA

The ABM-1310 single-patient EA is specifically for patients in the U.S. who previously participated in an ABM-1310 trial and continued ABM-1310 treatment when the trial ended.
This EA is only for patients with advanced BRAFv600 mutant solid tumors including primary brain tumors.

Available Therapies via Single-Patient EA

AC Immune SA, a clinical-stage biopharmaceutical company, is a global leader in developing precision medicine for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and certain rare indications.

AC Immune has determined that given the stage of development of its products, its investigational drugs should be studied in patients as part of controlled clinical trials designed to obtain data on safety and efficacy that may be used to support approval of the product and subsequent wider accessibility to patients. We encourage patients to speak with their physicians and to participate in the available clinical trials. At this time, AC Immune cannot provide investigational drug outside of clinical studies. The company will reevaluate this policy when sufficient safety and efficacy information has been obtained in controlled clinical trials.

Single-Patient EA Policies/Criteria

Our EA policy will apply to those DMG patients who exhausted standard of care and can't find appropriate trials to participate. We encourage a patient's provider (pediatric neuro-oncologist) to reach out to us so that we can discuss the case with the provider before an EA decision can be made based on the clinical data we collected so far and the information from each patient.

 

Available Therapies via Single-Patient EA

ACT001 for DMG treatment

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

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

Pre-Approval Access Principles:

a properly licensed treating physician requests the use of an argenx unapproved product to treat a patient that is seriously ill, has no alternative treatment options and cannot enroll in a clinical trial;

the requested product should be in active clinical development, and it is being requested to treat an indication for which there is positive data from a pivotal or registrational study;

there is sufficient scientific evidence to demonstrate that the benefits of the investigational medicine outweigh the risks;

the patient profile and requested treatment regimens are aligned with those from current registrational studies;

the request is neither tied to a commercial interest nor associated with any promotional activities;

providing the IMP for the requested use will not interfere with the initiation, conduct, or completion of clinical trials;

if approved, argenx will provide IMP and adhere to all local laws and regulations.

Available Therapies via Single-Patient EA

An Expanded Access Program for Efgartigimod Treatment in Patients With Generalized Myasthenia Gravis
Brief Summary
This expanded access protocol applies to patients with gMG who are not enrolled in an ongoing clinical trial. The aim of the trial is to provide patients with generalized myasthenia gravis (gMG), who are ineligible to participate in a clinical trial, access to efgartigimod treatment before regulatory approval. There are country-specific protocols and also individual use PAA. Recruitment for the treatment protocol in US is now closed (ARGX-113-EAP-2101) and in countries where reimbursement is obtained.

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

Overview

1. The decision to seek access for treatment utilizing the EAP must be initiated by the patient’s physician. Ascendis Pharma will not incentivize the healthcare provider (HCP) in any way for accessing any Investigational Treatment. The HCP will have sole responsibility and accountability for the clinical management and treatment of the patient.
2. Compliance Considerations:

• Requests for EAP use must comply with local standards of care, along with the laws and regulations that govern the use of any Investigational Treatment.
• An HCP request to access EAP on behalf of a patient may entail disclosure of personal patient medical information for assessment by Ascendis Pharma in order to assess eligibility. All patient information must be redacted so that no personally identifiable information is transmitted.
• Requests require both HCP to initiate the request as well as signed consent by the patient.

Criteria for Evaluating Individual Access

HCP Criteria and Responsibilities:

Must be currently licensed in his/her respective jurisdiction and must not ever have been convicted of a criminal offence relating to any therapy, food, or cosmetic law.
HCP must not be on the current FDA debarment list or that of any of the relevant local health authorities in the relevant jurisdiction.

Provide all necessary paperwork to Sponsor for routing to an Investigational Review Board (IRB) or Ethics Committee (EC) as appropriate in their relevant health authority’s jurisdiction.

The HCP must agree to oversee the patient’s treatment and supervise administration of the Investigational Therapy in accordance with Ascendis Pharma’s defined access criteria and under appropriate regulatory and ethical standards including those set by regulatory authorities and IRBs/ECs.

The HCP must agree to obtain informed consent and agreement for treatment use of the Investigational Therapy, and collection, analysis, publication, and transfer of any patient data as appropriate from the patient or his/her legal guardians.

The HCP must agree to maintain and release to regulatory authorities all treatment records and data as required by local regulations.

The HCP must agree to report adverse events and provide patient level safety data as required by local health authorities and per the agreement with Ascendis Pharma

Patient Eligibility Criteria:

1. A patient may obtain access to an Investigational Therapy for treatment use via EAP only through a licensed HCP.
2. Generally, patient eligibility for consideration of treatment with Investigational Therapy include, but not limited to:

• Patient must be diagnosed with either a serious, debilitating, or life-threatening medical condition.
• A clearly documented biological or clinical rationale should support the potential clinical benefit to the patient.
• Suitable treatment options are not available to treat the ongoing condition OR the patient has a documented attempt at current standard of care but has not responded to treatment.
• The patient does not have an option to participate in a relevant Ascendis sponsored clinical trial because of non-eligibility or the trial is not conducted in the patient's region.
• The patient must have continued access to the HCP for ongoing care.

Request Process

Requests for EAP must be made by the patient’s treating physician.

Available Therapies via Single-Patient EA

Palopegteriparatide (also known as TransCon PTH) for patients with hypoparathyroidism

Available Therapies via Single-Patient EA

This program is not available in the United States of America. 

 

Olverembatinib is a novel third generation BCR-ABL inhibitor that can effectively target a spectrum of BCR-ABL mutants, including the T315I mutation. Olverembatinib is approved in China for the treatment of adult patients with tyrosine kinase inhibitor (TKI)-resistant chronic phase chronic myeloid leukemia (CP-CML) or accelerated-phase CML (AP-CML) harboring the T315I mutation as confirmed by a validated diagnostic test. 

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

Participation in Atsena Therapeutics’ clinical trials is currently the only pathway for patients to gain access to our investigational therapies. We are not currently offering expanded access to any of our investigational therapies.

Single-Patient EA Policies/Criteria

At this time, participation in one of our Attralus clinical trials or investigator-sponsored clinical trials is the only way to access any Attralus investigational products. For more information regarding clinical trials with Attralus investigational therapies or diagnostics, please visit www.clinicaltrials.gov.

Expanded access requests will be referred to study investigators to determine whether a patient is eligible for any ongoing clinical trials. If a patient does not qualify for one of our ongoing clinical trials, referrals may also be provided to patient organizations and/or academic centers who may be able to identify other appropriate clinical trials or approved treatment options.

Available Therapies via Single-Patient EA

None at this time.

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

At our current stage of development, Avenge Bio does not have an active expanded access program that allows patients to have access to our investigational products prior to FDA approval. Avenge Bio may revise this expanded access policy at any time as we advance the development of our product and will make available the specific requirements to access the program.

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

At this time, Avobis Bio is offering an Expanded Access Program on a limited basis pursuant to Avobis’ Expanded Access Protocol. To ensure the safety of patients, we have developed criteria to evaluate applicable Expanded Access Program requests. Any such requests must be in accordance with laws and regulations and our internal policies and procedures. Further, we cannot guarantee access to any investigational drug by any individual patient. FDA guidelines note that Expanded Access may be appropriate when the below criteria are met:
1. Patient has a serious disease or condition, or whose life is immediately threatened.
2. There is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition.
3. Enrollment in a clinical trial is not possible.
4. Probable patient benefit justifies the probable risks of treatment with investigational drugs.
5. There is sufficient evidence of safety and effectiveness to support the use of the investigational drug.
6. Providing the investigational medical product will not affect the investigational trials.
7. The Expanded Access request is made by a physician who is qualified and licensed in accordance with State law and who is knowledgeable in the treatment of the disease or condition.

A treating physician must initiate an Expanded Access request. If you are a patient or caregiver, please reach out to your doctor to assist you in this process. Physicians should send Expanded Access requests to expandedaccess@avobisbio.com. The physician requesting Expanded Access must provide specific information supporting the request, including:
1. A copy of the requesting physician’s curriculum vitae (CV).
2. A copy of the physician’s current medical license, which must be in good standing in the state where the therapy will be administered. By submission of a medical license, the requesting physician attests they are not under any investigation or threatened loss of privileges to practice.
3. Relevant patient history, excluding any individually identifiable health information or protected health information (PHI).
4. Scientific rationale supporting the potential benefits of the investigational product based on available clinical and non-clinical data.
5. Statement that there is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the patient’s disease or condition.
6. Statement that the patient has exhausted all approved therapy options, or that the patient is no longer responsive to or able to tolerate such therapies.

We will acknowledge receipt of a physician’s request within five business days.
Medical and scientific professionals at Avobis Bio who are familiar with the available data for the investigational product will evaluate all applicable requests on an individual basis in accordance with the criteria above, pursuant to Avobis Bio’s Expanded Access IND and using the scientific evidence available at the time of the request and other information relevant to patient safety to aid their evaluation.
Posting this policy by Avobis Bio does not guarantee access to any specific investigational drug for any individual patient. We encourage patients to speak with their physicians about the possibility of enrolling in clinical trials in order to gain access to investigational therapies. Additionally, patients can search www.clinicaltrials.gov for information regarding clinical trials. If you have questions about our Expanded Access Program, please contact us at expandedaccess@avobisbio.com

Available Therapies via Single-Patient EA

AVB-114 is an autologous investigational product under evaluation in Seeded Cells on Matrix Plug Treating Crohn's Perianal Fistulas (STOMP-II; a prospective, multicenter, randomized, blinded outcome assessment, controlled, add-on therapy phase II study) for the treatment of complex perianal fistulas in subjects with Crohn's disease. EAP-STOMP2 is an expanded access protocol for patients who had adipose tissue collected per the STOMP-II protocol, but who were subsequently unable to receive AVB-114 treatment due to screen failure or product manufacturing issues and thus withdrew from the study. Patients who withdrew from STOMP-II for other reasons will not be considered for EAP-STOMP2. This EAP will only be considered at Clinical Investigative Sites who enrolled subjects in STOMP-II.

Inclusion Criteria
In order to be eligible to participate in this EAP, an individual must meet all of the following criteria:
1. Provision of signed and dated ICF.
2. Adipose tissue previously collected per the STOMP-II protocol, but were
subsequently unable to receive AVB-114 treatment due to screen failure or product manufacturing issues and thus withdrew.
3. Diagnosed with Crohn’s disease and a target perianal fistula with one internal
opening and at least one external opening.

Exclusion Criteria
An individual who meets one or more of the following criteria will be excluded from participation in this EAP:
1. Suffer from any condition or illness that, in the opinion of the Investigator, would compromise patient safety.
2. Pregnant, trying to become pregnant, or are breast feeding. Women of childbearing potential (WCBP) and males who have sexual partners that are WCBP must agree to use an adequate method of contraception while participating in this EAP.
3. Known allergies or hypersensitivity to aminoglycosides.
4. History of clinically significant fat-directed autoimmunity.
5. Active, unresolved infection requiring parenteral antibiotics.
6. Unresolved abscess at time of treatment (if screening Pelvic MRI identifies an
abscess, it must be resolved before treatment).
7. Target fistula with any of the following characteristics (note that presence of a second fistula tract that is distinctly separate from the target fistula, or a target fistula with a single internal opening and multiple external openings is not exclusionary):
a. Target fistula has a branching blind ending sinus tract (no external opening) that investigator assesses to be at elevated risk of abscess formation if treated with AVB-114.
b. Target fistula is a genito-urinary fistula, including rectovaginal (i.e., fistulae that
transverse the vaginal canal).
c. Any anatomical limitation to successfully securing the fistula plug disk.