Amylyx Pharmaceuticals Receives CHMP Negative Opinion on its Conditional Marketi… – Press Release


  • Amylyx will seek re-examination of its Conditional Marketing Authorisation Application

Amylyx Pharmaceuticals, Inc. AMLX (“Amylyx” or the “Company”) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the application for conditional marketing authorisation of AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine [also known as taurursodiol]), under the trade name ALBRIOZA®, for the treatment of adults with amyotrophic lateral sclerosis (ALS) in the European Union (EU). Today’s update follows the Company’s May 2023 announcement that the CHMP was trending toward a negative opinion.

“We are confident in the strength of our CENTAUR trial data, which we believe meets the criteria for conditional approval. These data were also the basis of the full approval received from the U.S. Food and Drug Administration and the approval with conditions from Health Canada,” said Tammy Sarnelli, Global Head, Regulatory Affairs and Clinical Compliance at Amylyx. “We disagree with the CHMP’s opinion and will request a formal re-examination procedure of the current Marketing Authorisation Application (MAA).”

CENTAUR 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 overall survival in a longer-term post hoc analysis. 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.

The CENTAUR data were published in peer-reviewed medical journals, including the New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry.

“We will continue to…