Sarepta Therapeutics has suspended shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, to nonambulatory patients following a second reported death due to acute liver failure.
The biotech company, based in Cambridge, Mass., also paused dosing in its Envision clinical trial, a global study evaluating Elevidys in older ambulatory and nonambulatory individuals, according to a June 15 news release from the company. The FDA agreed with the pause, which Sarepta said will allow time to evaluate a revised immunosuppressive regimen.
Elevidys was first approved by the FDA in June 2024 for certain patients with Duchenne muscular dystrophy, a rare genetic disorder that progressively weakens muscles. Elevidys remains the only approved gene therapy for the disorder.
The first patient death was reported by Sarepta in March 2025 when a young man experienced acute liver failure after receiving the therapy. The patient had also been treated with cytomegalovirus injections, which can damage the liver and may have contributed to the fatal outcome.
A study published April 2025 in Gene Therapy, suggests that the body’s natural immune response might undermine the safety and effectiveness of gene therapies such as Elevidys. Researchers from the University of Portsmouth speculated that dystrophin, the protein restored by the therapy, could be recognized as a foreign antigen by the immune system, which could erode therapeutic benefit and increase toxicity.
Sarepta said it is convening with an independent panel of experts in Duchenne and liver health to evaluate a proposed immunosuppression regimen. More than 900 individuals have received Elevidys through clinical trials and commercial use.
