The US Food and Drug Administration has updated a warning about the safety of Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD), and restricted its use. This follows reports earlier this year of fatal acute liver failure in certain non-ambulatory, male, paediatric patients taking the drug. Following the incidents, the developer, Sarepta Therapeutics Inc, voluntarily paused distribution of Elevidys to this patient group. This has now been formalised in a notice issued on 14 November. The new label includes a warning about the risks of serious liver injury and acute liver failure and limits the indication to ambulatory DMD patients. Patients eligible for treatment can be four years of age and older with a confirmed mutation in the DMD gene. An indication covering non-ambulatory patients has been removed.
DMD is a progressive, genetic disease caused by a gene mutation that can lead to the dysfunction or absence of the dystrophin protein. Elevidys is intended as a one-time gene transfer therapy which carries instructions for the delivery of a functional shortened dystrophin protein into muscle tissue. It was first approved in the US in 2023. The new label also requires the developer to conduct a postmarketing observational study to further assess the risk of serious liver injury. Separately, the European Medicines Agency declined to recommend the drug in an opinion issued on 24 July 2025.
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