Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys, is encountering significant regulatory and safety challenges in both Europe and the U.S. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended against its approval, citing failure to demonstrate efficacy in improving motor functions in children aged 4 to 7. The FDA has also requested Sarepta to pause shipments in the U.S. due to emerging safety issues, including multiple patient deaths. Regulatory authorities are pushing Sarepta for additional studies to confirm the therapy's safety, possibly involving dose adjustments and further analyses. The controversy has left patients and families in limbo, with mounting scrutiny threatening the drug's market future.