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# Amylyx Pharmaceuticals NDA for ALS Drug
- URL: https://www.fdaweb.com/amylyx-pharmaceuticals-nda-for-als-drug/
- Published: 2021-11-02T12:00:00.000Z
- Updated: 2026-09-14T17:21:03.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5150498

Amylyx Pharmaceuticals has submitted an NDA for AMX0035 (sodium phenylbutyrate and taurursodiol), indicated for treating amyotrophic lateral sclerosis (ALS). The submission is based on data from the CENTAUR trial, a placebo-controlled study evaluating 137 people with ALS. In this study, participants receiving AMX0035 had “statistically significant slowing of functional decline at the end of the six-month randomized phase as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS,” according to the company.

An interim survival analysis conducted in all randomized participants from CENTAUR showed a 44% lower risk of death compared to those who started on placebo during the placebo-controlled phase, Amylyx says. Median survival duration through the open-label long-term follow-up phase was 25.0 months for those on active therapy compared with 18.5 months for those on placebo.

AMX0035 is designed to reduce neuronal death and dysfunction, according to Amylyx, adding that it works by targeting the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.