Sarepta, FDA and gene therapy
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After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, noting the need to maintain a good working relationship with the regulator.
The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due to safety concerns.
The FDA will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following two deaths tied to the product.