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Our Commitment

If you are living with a serious and life-threatening neurodegenerative disease, we understand and share your urgency.

At Amylyx, we are committed to bringing potential new therapies to people living with neurodegenerative diseases as quickly as we can. To achieve this goal, we must follow each country’s rules, laws, and pathways set by regulatory agencies such as Health Canada, the U.S. Food and Drug Administration (FDA), and the European Medicines Agency (EMA), and other government agencies.

Use of Investigational Drug

As we study our investigational drug in clinical trials, we may be allowed to provide it to some people with serious progressive neurodegenerative diseases to use, even if they are unable to participate in a clinical trial.

Specifically, in cases where clinical trial participation is not an option and a person has exhausted other available treatment options, regulatory agencies may grant permission for Amylyx to provide a person access to an investigational drug prior to its approval. This use of an investigational drug pre-approval is often referred to as early access, but may be referred to by other names depending upon the naming convention used by a country’s regulatory agency. 

Our mission is to work with doctors, advocacy groups, people impacted by neurodegenerative diseases, and regulatory agencies to find the right time to provide early access to our investigational drugs if appropriate. 

Amylyx does not plan to provide early access to an investigational drug outside of the disease it was studied for in clinical trials.

It is important to remember that investigational drugs have not received regulatory agency approval. Because of this, the full potential of their risks and benefits are not known. Doctors and patients should consider all possible benefits and risks when seeking access to an investigational drug.

AMX0035 Access Programs

AMX0035 Early Access Programs in ALS by region

AMX0035 is an investigational drug being studied in the treatment of ALS.  It is a combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine)

At this time access to PB and TURSO is available commercially for people living with ALS. There is no access program for other serious and life-threatening neurodegenerative diseases. Please contact medinfo@amylyx.com with additional questions.

At this time access to PB and TURSO is available commercially for people living with ALS. There is no access program for other serious and life-threatening neurodegenerative diseases. Please contact medinfo@amylyx.com with additional questions.

Amylyx is conducting a Phase 3 clinical trial with participants from Europe and the United States to further evaluate the safety and efficacy of AMX0035 in ALS. The latest information available is at www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT05021536), www.clinicaltrialsregister.eu (EudraCT Number: 2021-000250-26), and www.AmylyxALSTrial.com.

In cases where participation in a clinical trials might not be an option for patients living with ALS, regulators/health authorities may grant permission for us to provide treating physicians with access to an investigational drug pre-approval for their eligible patients. Amylyx continues to work with regulatory agencies, physicians, people living with ALS and the broader ALS community to understand options, timing and scope for potential early access to investigational drugs in Europe. However, the timing and scope of such access cannot interfere with the conduct of the ongoing Phase 3 PHOENIX clinical trial or other aspects of development that could impact regulatory agency approval and access to the broader ALS community. We will share updates with the ALS community as and when we are able to do so.

On October 5, 2023, the Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM), the national authority for authorizing medical products in France, granted “accès compassionnel” (compassionate access) to AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine [also known as taurursodiol]), for eligible people living with amyotrophic lateral sclerosis (ALS) following discussions with Amylyx. For more information from ANSM, including eligibility criteria, please visit https://ansm.sante.fr/documents/reference/referentiel-des-specialites-en-acces-derogatoire.

At this time, access to Amylyx’ drug for the treatment of ALS is not available outside countries where it is approved (i.e., Canada, United States) or in Europe where access is currently limited to those participating in the Phase 3 PHOENIX clinical trial. For more information on PHOENIX, please click here. Please contact medinfo@amylyx.com with any questions.

Amylyx Clinical Trials

If you're interested in enrolling in an Amylyx clinical trial, talk to your doctor or care team to learn more

You may be able to join a clinical trial for our investigational drug if you qualify. Your doctor can help you decide if a clinical trial is right for you. To learn more about Amylyx clinical trials, visit amylyxalstrial.com and www.clinicaltrials.gov.

For questions about our policy on access to investigational drugs, please contact us at:

Medinfo@amylyx.com
(877) 374-1208
Monday-Friday, 9:00 AM-7:00 PM ET

We will work to respond to email requests
within 5 business days.

We reserve the right to update our policy at any time and will post any policy updates on this website as they become available.

 

Medinfo@amylyx.com
(877) 374-1208
Monday-Friday, 9:00 AM-7:00 PM ET

We will work to respond to email requests
within 5 business days.

We reserve the right to update our policy at any time and will post any policy updates on this website as they become available.

Glossary

Clinical Trial: For a new drug to be approved for use, companies like Amylyx are required to conduct research studies to evaluate the safety and efficacy of their investigational drug and submit the results to regulatory agencies. To participate in a trial, you must meet certain criteria. For those patients that meet the criteria of a clinical trial, participation comes with certain risks and these risks need to be discussed with your doctor.

Investigational Drug: A medicine that is being studied in a clinical trial. In most cases, investigational drugs may not be available to use outside of a clinical trial or other special authorization by a regulatory agency.

Early Access Program: In cases where participation in a clinical trial is not an option and a person has exhausted other available treatment options, regulatory agencies may grant special permission for Amylyx to provide patient access to an investigational drug prior to its approval.

This use of an investigational drug prior to regulatory agency approval is often referred to as early access, but may be referred to by other names, such as expanded access or compassionate use depending upon the naming convention used by a country’s regulatory agency.

Named Patient Program: In Europe, this program provides access to drugs that are approved and commercially available in one or more countries, other than the person’s home country.

Regulatory Agency: A government organization that decides how and when an investigational drug can be studied, approved and made available for use or authorized for early access.

Regulatory Processes: The series of steps and reviews required by a Regulatory Agency to approve a drug for use.