FDA extends NDA review for ALS drug

The FDA has extended its new drug application review of AMX0035, a combination of sodium phenylbutyrate and taurursodiol for the treatment of ALS, Amylyx Pharmaceuticals Inc. announced in a press release.

The extension was granted to allow more time to review additional data analyses from the company’s clinical studies of the drug. The FDA had originally granted an NDA for the oral, fixed-dose therapeutic in December 2021 following positive results from the CENTAUR study, a multicenter phase 2 clinical trial of 137 ALS patients that involved a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase. The trial met its primary efficacy endpoint of reducing functional decline as measured by the ALS Functional Rating Scale-Revised.

The FDA determined the updated information constitutes a major amendment to the NDA, resulting in an extension of the prescription drug user fee act date to Sept. 29, 2022.

“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,” Justin Klee and Joshua Cohen, co-CEOs and co-founders of Amylyx, said in the release.