After Initial Skepticism FDA's Adcomm Backs Amylyx's Amyotrophic Lateral Sclerosis Hopeful

  • The FDA Peripheral and Central Nervous System Drugs Advisory Committee voted (7 yes and 2 no) that the available evidence of effectiveness is sufficient to support the approval of Amylyx Pharmaceuticals Inc's AMLX AMX0035 for amyotrophic lateral sclerosis (ALS), just months after voting against the drug in a first adcomm.
  • The committee had previously voted against AMX0035 in March, deciding that the single randomized Centaur trial was not robust enough to prove efficacy, despite meeting its primary endpoint.
  • Related: FDA Again Highlights Lacking Data From Amylyx's Amyotrophic Lateral Sclerosis Candidate.
  • Amylyx also presented a post-hoc analysis comparing patients' progression in the AMX0035 group to their progression rates before the study and biomarker data from an Alzheimer's study to show the drug's effects on neurodegeneration.
  • Canada granted AMX0035 a conditional approval earlier this year, contingent upon a successful Phase 3 study.
  • AMX0035's PDUFA date is September 29.
  • Citigroup maintains Buy on Amylyx Pharma, raising the price target to $48 from $35.
  • Price Action: AMLX shares are up 63.20% at $29.22 on the last check Thursday.
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