ALS Drug Slows Progression, Petition for FDA Approval

The New England Journal of Medicine (NEJM) has published the results of a phase 2 trial (known as CENTAUR) of the compound AMX0035, produced by Amylyx, a Massachusetts-based pharmaceutical company. The results of the trial were very promising – people with ALS receiving this drug experienced a significantly slower decline in disease progression, compared to those on a placebo. Additionally, this drug shows to be safe and well-tolerated indicating a positive benefit and risk consideration for people with ALS.

The ALS Association and I AM ALS have now issued a petition calling on the drug company and the FDA to make the treatment widely available as soon as possible. The ALS Association and I AM ALS are asking that Amylyx and the FDA move with urgency to ensure this drug is a treatment option for all people living with ALS.

The ALS Association committed $750,000 to Amylyx in funding for this research, and $1.4M to the NEALS clinical trial consortium to support the trial. These grants were made possible through the ALS Ice Bucket Challenge. The trial included 137 people with ALS and was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium. All of the participants enrolled in the trial were diagnosed with sporadic or familial ALS within the previous eight months and had a rapid progression of the disease.

With a typical survival time of between 2 to 5 years, people with ALS cannot wait for the full experimental process of this drug to continue. In light of the substantive data of AMX0035’s effectiveness and safety, it has been requested that the FDA and Amylyx to work together to bring this to people with ALS as soon as possible by approving the drug prior to a Phase 3 trial. To access and sign the petition, visit www.als.org/petition.

Sign the Petition Today!