Sarepta Therapeutics has voluntarily paused shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following FDA requests amid safety concerns linked to acute liver failure and patient deaths. Roche, responsible for marketing Elevidys outside the U.S., is also halting shipments in certain markets reliant on FDA approval. The FDA and Sarepta are engaged in a regulatory standoff after Sarepta initially resisted halting shipments. The FDA highlighted risks including three patient deaths apparently due to liver failure associated with the gene therapy vector. Sarepta aims to collaborate with the FDA on label updates and safety reviews while seeking to maintain supply for ambulatory patients.