The FDA faces scrutiny amid complex regulatory challenges highlighted by Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy safety concerns, including cases of acute liver failure resulting in patient deaths. Sarepta reported zero deaths in ambulatory patients with positive risk/benefit profiles, but non-ambulatory patients remain vulnerable. Additionally, Schrödinger discontinued its CDC7 inhibitor SGR-2921 after two patient deaths in an early leukemia trial. These developments underscore ongoing tensions in rare disease drug oversight and clinical safety evaluation.