Multiple sources report that FDA Commissioner Marty Makary sought to influence the agency’s review process by advocating for rejection of KalVista Pharmaceuticals’ investigational rare disease drug. This unprecedented intervention raises concerns about regulatory impartiality under current FDA leadership. Concurrently, FDA scrutiny intensifies on Sarepta Therapeutics’ Duchenne muscular dystrophy therapy following patient deaths. The developments underscore challenges in balancing accelerated drug approvals and safety oversight amid organizational shifts at the FDA.