At Amylyx, we are committed to bringing all medicines shown to be safe and effective to people living with ALS or other 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.
UPDATE FOR PEOPLE LIVING WITH NEURODEGENERATIVE DISEASES, CAREGIVERS, AND FAMILY
Understanding Our Policy on Access to Investigational Drugs
- Amylyx's policy on access to investigational drugs
- How to get in touch with Amylyx about its Early Access Programs
- Specific steps to take to get more information
Amylyx is dedicated to developing therapies for patients with ALS and other neurodegenerative diseases as quickly as possible
We believe the best way to get helpful medicines to the most people who are qualified to receive it is to move through regulatory processes efficiently, following each country’s rules
In some cases, we may be able to provide an investigational drug for use even if is still being studied
For inquiries about our policy on access to investigational drugs, please contact us here
AMX0035 Access Programs
AMX035 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)
For information about access to Amylyx’s investigational drug for ALS in the US, please talk to your ALS doctor or care team to see if you are eligible for the US Early Access Program, which will provide access to our investigational drug for people who are not eligible for on-going clinical trials. For more information about the Early Access Program visit www.clinicaltrials.gov.
Amylyx is also conducting a Phase 3 clinical trial in Europe and the United States to build upon the evidence supporting AMX0035 for the potential treatment of 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.
For information about access to Amylyx’s investigational drug for ALS in Canada, please contact a doctor affiliated with the Canadian ALS Research consortium (CALS) or visit https://als.ca/support-services/other-provinces/
Amylyx is conducting a Phase 3 clinical trial in Europe and the United States to build upon the evidence supporting AMX0035 for the potential treatment of 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.
At this time, access to Amylyx’s investigational drug for ALS is not available in Europe. Access in Europe is currently limited to those participating in clinical trials, and requests for compassionate or named patient use in individual patients are not currently being considered. Amylyx will be working with regulatory agencies, doctors, 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 we have them.
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