ALS Drugs: Second US FDA Meeting On AMX0035 Gives Amylyx A Chance To Capitalize On Recent Events
Company is coming into its second panel meeting with new data analyses from the CENTAUR trial, advisory committee experience under its belt, and a recent Health Canada approval. In an interview with the Pink Sheet, co-CEOs Cohen and Klee highlight clinical outcomes data with AMX0035 and say they are not too worried about the lack of effect on neurofilament biomarker.
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Regulatory Flexibility: US FDA Approves Amylyx’s Relyvrio For ALS Despite ‘Degree Of Residual Uncertainty’
The single positive Phase II CENTAUR study, along with confirmatory evidence of benefit on long-term survival in the open-label extension, demonstrate substantial evidence of effectiveness, the agency says, acknowledging a level of uncertainty that is acceptable in the disease context.
Office of Neuroscience Director Billy Dunn touted FDA’s ability to withdraw approval for AMX0035 if subsequent data indicate a lack of substantial evidence of effectiveness. However, this provision appears to have been infrequently used and involves a potentially time-consuming hearing.
Recent and upcoming US FDA advisory committee meetings and a summary of topics covered.