Sarepta Therapeutics faces intensified regulatory scrutiny following multiple patient deaths linked to its Duchenne muscular dystrophy gene therapy, Elevidys. The FDA is actively investigating an 8-year-old boy’s death in Brazil and has requested a voluntary halt of Elevidys shipments in the U.S., with Sarepta suspending distribution temporarily. Concurrently, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) rejected Elevidys approval after study data failed to demonstrate efficacy in ambulatory children ages 4 to 7. Sarepta is expected to conduct further safety validation studies, potentially modifying dosing or manufacturing processes, under FDA direction. Parallel regulatory developments include Roche suspending shipments outside the U.S. and legislative debate on ongoing oversight. These events contribute to market uncertainty surrounding Elevidys and Sarepta’s strategic positioning in the Duchenne gene therapy space.