Following two reported deaths linked to Elevidys, its approved gene therapy for Duchenne muscular dystrophy, Sarepta Therapeutics has agreed to a black box warning on the drug’s label addressing risks of acute liver injury and failure. Shipments to non-ambulatory patients are paused while the company develops a new immunosuppressive regimen to mitigate liver toxicity, to be submitted for FDA review. The regulatory concerns and safety issues have driven Sarepta to restructure financially and strategically, focusing on manufacturing cost reductions and pipeline prioritization.