Sarepta Therapeutics has suspended shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, for non-ambulatory patients after two deaths from acute liver failure. The first patient death was reported in March, and both cases were related to the therapy’s side effect. Most children with Duchenne muscular dystrophy lose the ability to walk by adolescence.
The company is working with experts to develop an enhanced immunosuppressive regimen that could make Elevidys safer for non-ambulatory patients. It plans to discuss its proposal with the US FDA, aiming to minimize risks and ensure patient safety.
Source: https://www.statnews.com/2025/06/15/duchenne-sarepta-gene-therapy-elevidys-patient-death