Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys, confronts increasing regulatory challenges worldwide. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has rejected approval after studies failed to demonstrate improvement in patients' motor abilities. Meanwhile, the U.S. Food and Drug Administration (FDA) has requested Sarepta conduct new safety studies before allowing the therapy back on the market following concerns including patient deaths. This regulatory scrutiny has led Sarepta to pause shipments and restructure, impacting patient access and company operations significantly.