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Amylyx Pharmaceuticals Announces Outcome of FDA Advisory Committee Meeting on AMX0035 for the Treatment of ALS
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) announced today the outcome of the U.S. Food and
About this update from Amylyx Pharmaceuticals, Inc.
[{"type":"text","content":" CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAmylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) announced today the outcome of the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) meeting to review the Company’s New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).\n\nOn the question, “Do the data from the single randomized, controlled trial and the open-label extension study [Phase 2 CENTAUR trial] establish a conclusion that sodium phenylbutyrate/taurursodiol [AMX0035] is effective in the treatment of patients with amyotrophic lateral sclerosis (ALS)?,” the PCNSDAC voted 4 (yes) and 6 (no).\n\n“We remain confident in the data from the Phase 2 CENTAUR trial and the potential benefits of AMX0035 as a treatment option for people living with ALS,” said Jamie Timmons, M.D., Head of Scientific Communications of Amylyx. “We are motivated by the hundreds of people impacted by this devastating disease who shared their personal testimonies, both written and spoken, in conjunction with today’s meeting. We are also encouraged by the expert ALS physicians who shared their clinical perspectives. Our application is under review by the FDA, and we remain committed to pursuing its approval given the pressing need for new treatments for ALS.”\n\nAlthough 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 AMX0035 and is expected to make a decision on the approval of AMX0035 by June 29, 2022 under the Prescription Drug User Fee Act.\n\nALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death.\n\nAbout AMX0035\n\nAMX0035 is a proprietary oral fixed-dose combination of two small molecules: sodium phenylbutyrate (PB), which is a small molecular chaperone designed to reduce the unfolded protein response (UPR), preventing ...