Amylyx Pharmaceuticals has announced plans to submit a New Drug Application (NDA) for AMX0035 to the U.S. Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS). AMX0035 is a combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO) that was the subject of the CENTAUR trial, and the application for approval is a major step forward. Read their announcement.

The CENTAUR Trial of AMX0035 was led by Sabrina Paganoni, MD, PhD, Merit Cudkowicz, MD, MSc and the coordination center at the Healey Center for ALS. It was sponsored by Amylyx Pharmaceuticals and enrolled people with ALS across the country as part of the Northeast ALS Consortium, including at Mass General.  

Additional news will be forthcoming as Amylyx files their application with the FDA, and Drs. Cudkowicz and Paganoni will be participating in a series of webinars on the topic over the next few weeks.

I AM ALS Community Conversations with Amylyx Pharmaceuticals will be held on Wednesday, September 15 at 1:30 pm Eastern Time and Thursday, September 16 at 6:00 pm Eastern Time. Click here to register.

Northeast ALS Consortium (NEALS) will host a webinar to discuss the new drug application on Thursday, September 23 at 1:00 pm Eastern Time. Click here to register.