Sarepta Suspends Some DMD Gene Therapy Shipments Following Second Patient Death

Sarepta Therapeutics has temporarily suspended shipments of delandistrogene moxeparvovec-rokl (Elevidys), the company’s Duchenne muscular dystrophy (DMD) gene therapy, for non-ambulatory patients following a second reported case of acute liver failure resulting in death. Both fatal cases occurred in non-ambulatory individuals who received the gene therapy.

In response, Sarepta paused dosing in the ongoing ENVISION clinical trial, which is evaluating the therapy in older ambulatory and non-ambulatory patients with DMD. Additionally, the company is convening an independent panel of experts to evaluate an enhanced immunosuppressive regimen aimed at reducing liver toxicity risks among non-ambulatory patients. Sarepta will share the panel’s recommendations with the Food and Drug Administration, which is investigating the patient deaths, and has reported the event to global regulatory authorities.

Elevated liver enzymes and acute liver injury are known risks associated with AAV-based gene therapies, and delandistrogene moxeparvovec-rokl carries warnings for liver toxicity in its prescribing information. The therapy remains available for ambulatory patients under the current corticosteroid protocol.