The FDA is preparing to halt shipments of Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy due to safety concerns following multiple patient deaths from acute liver failure. Sarepta had already paused distribution for patients who are wheelchair-bound but continued supplying younger ambulatory patients until now. The agency is investigating the liver toxicity risks associated with Elevidys and a similar investigational gene therapy for limb-girdle muscular dystrophy with which a patient recently died. Sarepta’s CEO has faced criticism over delays in publicly disclosing these fatalities. Former FDA official Peter Marks and other experts have expressed support for the agency's cautionary approach, highlighting broader implications for the gene therapy field. This marks a major regulatory challenge for Sarepta’s pipeline and safety oversight in emerging AAV-based gene therapies.