Sarepta's Duchenne Therapy Sparked Fears
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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.
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular dystrophy over safety issues, even as patients and investors expressed growing concern about the company’s decision-making.
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.