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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Company seeks accelerated approval based on changes in neurofilament as a surrogate for efficacy; with controversy over FDA’s Aduhelm approval in Alzheimer’s still raging, agency is again faced with deciding whether biomarker data in a neurodegenerative disease with high unmet need are sufficient to justify approval in the face of a failed clinical endpoint study.
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.’