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Amylyx Pharmaceuticals Provides Update on Ongoing Regulatory Review of AMX0035 for the Treatment of ALS in the European Union
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced an update on the ongoing review
About this update from Amylyx Pharmaceuticals, Inc.
[{"type":"text","content":" CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAmylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced an update on the ongoing review of its Marketing Authorisation Application (MAA) for AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine [also known as taurursodiol]) for the treatment of adults with amyotrophic lateral sclerosis (ALS) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Following an oral explanation held at the May meeting of the CHMP, Amylyx was informed that the CHMP is trending toward a negative opinion on the application for conditional marketing authorisation of AMX0035.\n\n\n“We disagree with the current view expressed by the CHMP and remain confident in the data from the CENTAUR trial. Should a negative opinion ultimately be issued, we intend to request a formal re-examination procedure,” said Tammy Sarnelli, Global Head, Regulatory Affairs and Clinical Compliance at Amylyx.\n\n\nThe CHMP is expected to adopt a formal opinion on the MAA at its next meeting, which will be held June 19-22, 2023. If a negative opinion is received in June, the Company intends to request a formal re-examination of the opinion, which is an approximately four-month process.\n\n\nThe MAA for AMX0035 is based on data from the CENTAUR clinical trial, a randomized, multicenter, placebo-controlled trial in participants with ALS (n=137), which were the basis of the full approval received from the U.S. Food and Drug Administration (FDA) and approval with conditions by Health Canada. The trial met its prespecified primary outcome and AMX0035 is the first ALS therapy to demonstrate, in the same trial, both a statistically significant benefit in function, as well as an observed benefit on survival in a longer-term post hoc analysis. The CENTAUR data were published in the peer-reviewed medical journals the New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry. AMX0035 demonstrated a generally well-tolerated safety profile in the CENTAUR trial, with similar reported rates of adverse events and discontinuations in AMX0035 and placebo groups during the 24-week randomized phase; however, gastrointestinal events occurred with greater frequency (≥2%) in the AMX0035 group.\n\n\nALS affects approximately 29,000 people in...