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Sarepta Therapeutics has temporarily suspended the use of its gene therapy Elevidys in non-ambulatory patients with Duchenne muscular dystrophy (DMD) following a second patient death from acute liver failure. The decision, announced after the company disclosed the latest fatality, has prompted a pause in both commercial shipments and dosing in a major ongoing clinical trial.
According to Sarepta, both patients who died were non-ambulatory—meaning they relied on wheelchairs due to the progression of their disease. The first death, involving a 16-year-old boy, was reported in March. The company stated that both cases were linked to acute liver failure, a known potential side effect of gene therapies using adeno-associated virus (AAV) vectors.
Sarepta is now working with regulatory authorities and medical experts to evaluate an enhanced immunosuppressive regimen aimed at improving the safety of Elevidys for non-ambulatory patients. Shipments of the therapy for this group have been halted while these measures are reviewed and implemented. For ambulatory patients—those who can still walk—no changes to treatment protocols are currently proposed, and the standard use of corticosteroids and post-treatment monitoring remains in place.
Roche, Sarepta’s partner for international commercialization of Elevidys, has also discontinued the therapy for non-ambulatory patients outside the United States. Additionally, Sarepta has paused dosing in the ENVISION clinical trial, a global study serving as the confirmatory trial required by the U.S. Food and Drug Administration (FDA) for continued approval of Elevidys in non-ambulatory patients.
The FDA had previously granted accelerated approval for Elevidys for all DMD patients with a confirmed mutation in the DMD gene who are ages four and older, including non-ambulatory patients, contingent upon verification of clinical benefit in a confirmatory trial. The recent events have raised concerns among investors and the broader gene therapy community about the unpredictability of safety risks associated with these advanced treatments.
Sarepta plans to discuss the new safety measures and the impact of these developments with regulators, investigators, and the DMD patient community at upcoming conferences.
Disclaimer:
This article is based on publicly available information and corporate statements as of June 2025. The facts presented are subject to change as new data or regulatory actions emerge. The information is not intended as medical advice, and patients and caregivers should consult healthcare professionals for guidance on treatment options.
