ALS Drugs: A Long History Of Regulatory Twists At US FDA
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.
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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.
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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