Amylyx’ Second Chance Advisory Committee: Two Quasi-Precedents
US FDA’s plan to reconvene an advisory committee for a second look at Amylyx’ ALS drug AMX0035 is a remarkable twist in the ongoing review. It is essentially unprecedented, though there are two examples from the 1990s that offer some parallels.
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Amylyx’s ALS Drug Lacks ‘Confirmatory Evidence’ Needed To Support Approval On Single Study, FDA Says
New analyses on individual responders, survival and biomarker data are not independent evidence of AMX0035’s clinical effect in ALS, agency says in advisory committee briefing document. However, FDA’s framing of the issues, with an emphasis on regulatory flexibility and unmet medical need, could sway a favorable recommendation in its second panel review.
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.
The US FDA is set for two high-profile advisory committee meetings on potential ALS treatments before the end of 2022. Whatever the outcome, they will fit a pattern: when it comes to ALS treatments, there is very little about the regulatory process that feels routine.