Sarepta Halts DMD Trials After Teen Deaths

Sarepta Therapeutics has suspended shipments of its gene therapy Elevidys for non-ambulatory Duchenne muscular dystrophy (DMD) patients and paused dosing in its Phase III ENVISION trial after two non-ambulatory teenagers died from acute liver failure within three months. Both deaths, associated with the adeno-associated virus-based therapy, have intensified scrutiny of the FDA's expedited approval process for Elevidys and raised broader safety concerns about AAV gene therapies. Sarepta is collaborating with independent experts and regulators to develop enhanced immunosuppressive protocols before resuming treatment. Analysts have downgraded Sarepta’s stock and slashed price targets, citing significant risks to the therapy's commercial prospects and future sales. Elevidys remains available for ambulatory DMD patients and is the only FDA-approved gene therapy for the condition. The incidents are prompting industry-wide reassessment of AAV-based gene therapy safety and may influence future regulatory actions.