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Single-Patient EA Policies/Criteria

The goal of this program is to provide expanded access (i.e., before marketing authorization) to tiratricol as treatment for patients with monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome [AHDS]), who in their Treating Physician's opinion, could benefit from tiratricol and meet the eligibility criteria.

 

Available Therapies via Single-Patient EA

Therapy: tiratricol - currently and investigational drug for the treatment of monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome [AHDS].
 

Single-Patient EA Policies/Criteria

Last updated: February 2024

Ellipses Pharma Limited is a clinical-stage precision drug development biotechnology company focused on developing novel therapies for cancers. We conduct clinical trials to evaluate the safety and efficacy of investigational product candidates.
Ellipses encourages awareness of, and participation in, our clinical trials. For information regarding these trials, please visit clinicaltrials.gov and search for Ellipses Pharma.

Our current focus is to complete our ongoing clinical trial for EP0031 to demonstrate safety and efficacy and to obtain regulatory approval and provide widespread availability of EP0031.

Prior to regulatory approval by global regulatory authorities, patients gain access to investigational treatments, including Ellipses’ development candidates, by participating in clinical trials. In certain cases, when it is not possible for a patient to participate in a clinical trial, and where all other available medical options have been exhausted, a patient’s doctor may attempt to seek special access to an investigational product candidate for that patient outside of a clinical trial. This is known, among other terms, as expanded access or compassionate use.

For a patient to obtain access to an investigational product candidate through expanded access, the patient’s doctor, the drug’s sponsor and the regulatory authority in the doctor’s country of practice (e.g., FDA in the US or the MHRA in the UK etc) must all approve the use. Unlike the use of an investigational new drug in a clinical trial setting, the primary purpose of expanded access is to use the investigational drug for patient treatment purposes, rather than to gather data on safety, tolerability and effectiveness.

Currently, Ellipses Pharma does not offer an expanded access program. We believe that access to our investigational product candidates should be limited to controlled clinical trials until such time as their safety, tolerability and effectiveness have been determined and confirmed by regulatory authorities.

Additionally access to our development candidates before dose and dosing regimen have been optimized and safety and tolerability have been confirmed may not be in the best interest of patients.

Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials are the only safe and appropriate way to access our investigational product candidates.

In accordance with the 21st Century Cures Act this policy may be revised at any time and will be shared with the public on the Company website (ellipses.life) and the Reagan-Udall Foundation Expanded Access Navigator (navigator.reaganudall.org)
Should Ellipses Pharma decide to make its investigational medicinal products available on an expanded access basis, this policy will be updated with a hyperlink to the expanded access record on clinicaltrials.gov after that record becomes active.
If you have questions about Ellipses Pharma’s expanded access policy, please contact us at expanded.access@ellipses.life.

Ellipses Pharma Limited may revise this policy at any time.

Available Therapies via Single-Patient EA

Currently, Ellipses Pharma does not offer an expanded access program. We believe that access to our investigational product candidates should be limited to controlled clinical trials until such time as their safety, tolerability and effectiveness have been determined and confirmed by regulatory authorities.

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

Exciva is currently developing an investigational product (EXV-802) for potential treatment for agitation in people with Alzheimer’s Disease. Exciva understands that there may be individuals who are interested in access to EXV-802 outside of a clinical trial. At this stage of development, the only way to gain access to EXV-802 is by participating in a clinical study.

 

Single-Patient EA Policies/Criteria

Exelixis believes that the best way for a patient to have access to an investigational medication is through a clinical trial. Clinical trials, which may be sponsored by Exelixis or by another institution such as an academic medical center or a governmental body, enable rigorous systematic evaluation of a medication and allow for close monitoring of enrolled patients. Clinical trials, including eligibility criteria, are listed at ClinicalTrials.gov. Patients interested in learning if enrollment in a clinical trial is an option for them should consult with their treating physician.

Patients who are not eligible for enrollment in a clinical trial may be able to access an investigational medication via early access. Early access is only available in limited circumstances for certain types of patients for which no other treatment options exist. Provision of an investigational medication by Exelixis will always be done in accordance with applicable laws and regulations. At Exelixis, early access to an investigational medication outside of a clinical trial might be possible through an “Expanded Access Program,” a single-patient investigational new drug application submitted to the U.S. Food and Drug Administration, or via a “Named Patient Use” program in other countries.

The following are some of the general considerations for early access:

• For an Exelixis investigational medication to be made available to a patient via early access, the patient must be diagnosed with a serious or immediately life-threatening disease for which no satisfactory treatment alternative exists.
• The medication must be under clinical development by Exelixis, and the provision of the medication must not compromise the clinical development program (e.g., the initiation, conduct or completion of trials) or the potential regulatory approval of the medication.
• Exelixis must have adequate supply of the medication.
• Sufficient data must exist to support its use in the specific clinical condition from which the patient suffers, and adequate data must also exist to identify an appropriate dose for use in such patients.
• Overall, the potential benefit to the patient must outweigh the potential risks to the patient.

Patients who are interested in obtaining early access to an Exelixis investigational medication should discuss the matter with their treating physician. A request for early access must come directly from the physician and should be conveyed to Exelixis Medical Information at medinfo@exelixis.com or by calling 1-855-292-3935. Physicians who administer Exelixis investigational medications through early access are required to comply with all applicable laws and regulations and contractual conditions, including those relating to safety reporting.