this post was submitted on 23 Jun 2023

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First gene therapy for deadly form of muscular dystrophy gets FDA approval for young children (www.pbs.org)

submitted 1 year ago by alyaza to c/science

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[–] wjs018 2 points 1 year ago

So, Sarepta has multiple approved therapies that haven't reached positive clinical endpoints? How many times can the agency/drugmaker justify accelerated approval based on an unmet clinical need?

Imo, this just adds to the growing criticism of the agency's growing use of accelerated approvals without holding pharma companies to their post-approval obligations.