Post-FDA Talks, Amylyx Pursues Approval for ALS Drug

Following encouraging talks with the FDA in July, Amylyx Pharmaceuticals plans to submit a new drug application (NDA) for AMX0035, a compound designed to slow neurodegeneration in amylotropic lateral sclerosis (ALS) patients.

If approved, AMX0035 (sodium phenylbutyrate and taurursodiol) would be the first new ALS drug since 2019.

Amylyx said the FDA “expressed an interest in seeing data from an additional placebo-controlled clinical trial” before considering an NDA, so the company laid plans for a phase 3 trial based in the U.S. and Europe.

However, the agency also said it would “continue to discuss with Amylyx how regulatory requirements may be met in the most expeditious way possible,” the company said. Apparently, a July 15 pre-NDA meeting satisfied the FDA’s stipulations.