HIGH IMPACT – The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) reviewed Amylyx’s NDA for AMX0035 in ALS and voted that the current data do not support the efficacy for AMX0035 to treat ALS.

  • Amylyx’s FDA submission was based on a 24-week placebo-controlled study of 137 participants with ALS.
  • The panel voted 4 Yes 6 No and 0 Abstain 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 [AMX0035] is effective in the treatment of patients with amyotrophic lateral sclerosis (ALS)?
  • In response to FDA’s decision, Amylyx said it is confident in the trial data and the benefits of the drug as a treatment option for ALS.
  • The PDUFA date for the FDA’s priority review decision on the approval of AMX0035 is expected on June 29th, 2022.

inThought Analysis

The decision was in line with Monday’s briefing documents, where the FDA staff reviewers raised concerns over the drug’s trial data and efficacy. The tight vote against the drug contrasted with strong support from patients during the “open public hearing”, where several noted that both the FDA and Amylyx agree that AMX0035 “causes no material harm”. Amylyx recently initiated a larger trial in about 600 patients globally, but completion is not expected until 2024.


Source: Amylyx Press Release