Amylyx Will Submit ALS Drug For Approval In The Coming Months

Amylyx Pharmaceuticals, Inc. said on 15 September that it will submit a new drug application (NDA) to the US Food and Drug Administration “in the coming months” seeking approval for AMX0035 to treat amyotrophic lateral sclerosis (ALS), well before completion of its soon-to-be-initiated Phase III clinical trial. The FDA filing plan is a shift from just two months ago when the Cambridge, MA-based private company, which intends to commercialize its drug globally on its own, raised $135m to fund the Phase III study and file AMX0035 with Canadian and EU regulators.

Exactly when Amylyx could file in the US was unclear as recently as July, when the company announced its new financing, which followed an update in April noting that the agency wanted to see results from an additional placebo-controlled trial before receiving the NDA but was open to discussing an earlier filing. (Also see "Finance Watch: $135m In Venture Capital Will Fund Amylyx’s Late-Stage ALS Drug" - Scrip, 22 July, 2021