Sarepta Defies FDA, Keeps Shipping Gene Therapy | JQJO

Sarepta Therapeutics refused an FDA request to halt shipments of its gene therapy, Elevidys, following the death of a third patient. The FDA cited safety concerns after the deaths of three patients, all linked to liver injury, but Sarepta maintains that its scientific review shows no new safety signals for younger patients. The company plans to continue providing the drug to younger patients, despite the FDA's request and the significant stock drop following the announcement. The FDA can take action to remove the drug from the market, but the process is lengthy. Sarepta recently laid off 500 employees and added a warning to the drug's labeling.