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FDA staff skeptical of Sarepta’s Duchenne gene therapy, documents show

May 10, 2023

Food and Drug Administration scientists evaluating what could be the first gene therapy for Duchenne muscular dystrophy appear skeptical of the treatment’s benefit, adding to questions about its approval prospects days before a crucial regulatory meeting.

On Friday, the FDA will convene a panel of experts to discuss use of therapy, developed by biotechnology company Sarepta Therapeutics, for Duchenne patients who can still walk. Panelists will be asked to vote on whether an “accelerated” approval should be granted based on the therapy’s ability to help produce a potentially helpful protein called microdystrophin. Sarepta believes increases in this protein are “reasonably likely” to translate to clinical benefits.

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