FDA Advisory Committee meeting on AMX 0035 for the treatment of ALS gives a negative decision.- Amylyx Pharma
Amylyx Pharmaceuticals, Inc. announced the outcome of the FDA's Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) meeting to review the Company’s New Drug Application (NDA) for AMX 0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).
On the question, “Do the data from the single randomized, controlled trial and the open-label extension study [Phase II CENTAUR trial] establish a conclusion that sodium phenylbutyrate/taurursodiol [AMX 0035] is effective in the treatment of patients with amyotrophic lateral sclerosis (ALS)?,” the PCNSDAC voted 4 (yes) and 6 (no).
Although the FDA considers the recommendations of its advisory committees, the recommendations by the panel are non-binding. The final decision regarding approval of a pending NDA is made by the FDA. As previously reported, the FDA has granted Priority Review to the NDA for AMX 0035 and is expected to make a decision on the approval of AMX 0035 by June 29, 2022 under the Prescription Drug User Fee Act.
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