Cambridge, Massachusetts–based Amylyx Pharmaceuticals intends to submit a New Drug Application (NDA) to FDA for AMX0035 (sodium phenylbutyrate and taurursodiol) to treat amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease.
The company decided to file after a series of meetings with the FDA, including a pre-NDA meeting on July 15.
AMX0035 was the subject of the Phase 2 CENTAUR trial, which involved 137 participants with ALS. In that trial, the drug met its primary efficacy endpoint of slowing ALS based on the ALS Functional Rating Scale-Revised (ALSFRS-R).
A forthcoming Phase 3 trial will evaluate the safety and efficacy of the drug over 48 weeks.
Before reversing course, FDA had asked Amylyx to submit positive Phase 3 trial results before filing a New Drug Application.
To date, there are limited treatment options available for ALS. A 2020 paper in Neuropharmacology.
Concluded that the two drugs with demonstrated efficacy in treating ALS, Riluzole (Rilutek) and edaravone (Radicava), only “show only modest effects on disease progression.”
“AMX0035 has shown potential to provide those living with ALS and their families hope for the future,” said Dr. Sabrina Paganoni, principal investigator of the CENTAUR trial, in a statement.