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FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections

  1. Asher Mullard
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The FDA has granted accelerated approval to Sarepta’s gene therapy delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy (DMD). It is the first time the FDA has greenlit a gene therapy using accelerated approval. This regulatory pathway is intended to provide earlier access to therapies that have an effect on surrogate endpoints that might predict clinical benefit, and requires drug developers to provide confirmatory evidence of efficacy. But regulatory documents show that agency reviewers were divided on the case for the therapy’s efficacy.

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Nature Reviews Drug Discovery 22, 610 (2023)

doi: https://doi.org/10.1038/d41573-023-00103-y

Updates & Corrections

  • Update 27 June 2023: The article has been updated to highlight an FDA memo and details on the clinical trials have been shortened.

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