The ALS Association, I AM ALS Call On FDA, Amylyx to Make AMX0035 Available to People with ALS
In response to the New England Journal of Medicine publication of encouraging clinical trial results showing that Amylyx Pharmaceuticals’ AMX0035 brought statistically significant benefit to people living with ALS, The ALS Association and I AM ALS immediately called on the drug company and the FDA to make the treatment widely available as soon as possible. Data from the trial (known as CENTAUR), indicates the drug met its pre-specified, primary outcome and represents a significant development for the ALS community.
In a petition made public this morning, The ALS Association and I AM ALS asked that Amylyx and the FDA move with urgency to ensure this drug is a treatment option for all people living with ALS. Specifically, it asks that Amylyx submit a New Drug Application (NDA) for AMX0035 to FDA for approval, and that the FDA review and approve that NDA swiftly. It asks that the FDA require post-approval studies of AMX0035 on safety and effectiveness in the real world. Finally, it asks that Amylyx makes AMX0035 available through expanded access until it is brought to market. The public can read and sign the petition www.als.org/petition.
“The data on AMX0035 is encouraging and we are optimistic that the drug can help people with ALS,” said Fred Fisher, President and CEO of the Golden West Chapter of The ALS Association. "We look forward to working with Amylyx, the FDA and the ALS community to make this treatment an option for everyone with ALS as soon as possible.”
Fisher added, “The FDA has the statutory authority to be flexible when reviewing drugs that meet criteria for safety and show promise for people with terminal diseases. This authority, combined with the ALS community’s expressed willingness to accept risk for potential treatment benefit, should compel the FDA to work quickly and cooperatively with Amylyx to make AMX0035 available to the entire ALS community without delay.”
“I AM ALS is committed to getting treatments and cures to people living with ALS and the success of Amylyx's Phase 2 study demonstrates real progress toward that goal. The therapy has shown significant results in slowing progression of the disease and provides real hope,” I AM ALS CEO Danielle Carnival said. “Every day this community fights for the system, care and treatments it deserves. We ask the FDA and Amylyx to help us make that promise real by bringing this treatment to all those living with ALS."
In its news release detailing the findings published in the New England Journal of Medicine article, Amylyx leadership says that AMX0035 was well-tolerated and decreased the rate of decline in the Revised ALS Functional Rating Scale (ALSFRS-R) compared to placebo. The trial included 137 people with ALS and was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium.
The ALS Association committed $750,000 to Amylyx in funding for AMX0035 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. ALS Finding a Cure also supported this study.
Through a standard repayment clause, the Association could recoup up to 150% of its research investment, which it will reinvest in additional research.