Amylyx' ALS drug accepted for FDA priority review

US CNS-focussed biotech Amylyx Pharmaceuticals yesterday revealed that the US Food and Drug Administration has accepted for review its New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol [TURSO; also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).

The FDA has granted Priority Review and assigned a Prescription Drug User Fee Act date for AMX0035 of June 29, 2022, the target date by which the FDA intends to complete its review and take action on the NDA. The agency noted that it is currently planning to hold an advisory committee meeting to discuss the application. Amylyx additionally is preparing to submit an Expanded Access Program (EAP) to the FDA for launch in the USA in the coming months for patients who are ineligible for participation in the global Phase III PHOENIX clinical trial.

Could be first new ALS drug approval since 2017