STAT May 10, 2023
On Friday, the Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee will meet to discuss the Biologics License Application for Sarepta Therapeutics’ gene therapy delandistrogene moxeparvovec for the treatment of Duchenne muscular dystrophy. The late decision by the FDA to reverse course and hold an advisory committee to get expert input into the drug’s review occurred after concerns were apparently raised that the FDA was leaning toward rejecting the drug. This would have been a devastating blow for the boys living with Duchenne today, who have only a short window to get optimal benefit. Any further delay would harm their futures.
While holding this advisory committee meeting doesn’t ensure a different outcome, it is an important opportunity...
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