FDA Extends PDUFA Date of Amylyx Investigational ALS Drug

The FDA has pushed back its user fee decision date for Amylyx Pharmaceuticals’ New Drug Application (NDA) for its investigational drug, AMX0035, for treatment of amyotrophic lateral sclerosis (ALS) by three months, from June 29 to Sept. 29.

The agency said it needed more time to review additional analyses of clinical data submitted by the company, which the agency deemed to be a major amendment to the NDA.

In a March 30 meeting, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 against recommending AMX0035 for patients with ALS. But the ALS Association said the drug has been proven safe and it is hoping that the FDA will approve the treatment despite the expert panel’s reservations.

“We are confident in the potential of AMX0035 to help people living with ALS and other neurodegenerative diseases, and we continue to work closely with the FDA as they complete their review,” said Justin Klee and Joshua Cohen, the company’s co-CEOs and co-founders.