For Amylyx AMX0035 Panel Review, US FDA Relied Heavily on Temporary Voting Members
Peripheral and Central Nervous System Drugs Advisory Committee has held two of FDA’s most closely watched meetings in recent years, but there was little overlap between the panel that reviewed Amylyx’s ALS drug and the one that evaluated Biogen’s aducanumab 16 months earlier.
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Six of 10 advisory committee members say Phase II CENTAUR trial cannot stand on its own to support approval at this time. However, patients and advocates say thousands will die if they have to wait for the Phase III PHOENIX trial to complete in 2024 before the drug becomes widely available.
US FDA’s Billy Dunn says that if the drug had shown a positive effect on plasma neurofilament heavy chain, it ‘would have provided important contextual and supportive information of an ostensibly beneficial effect on the clinical measure.’
Citing numerous concerns about design of the Phase II CENTAUR trial and statistical robustness of the results, agency reviewers question whether a single study provides substantial evidence of efficacy for approval of AMX0035.