Insider reports reveal that the FDA commissioner allegedly pushed against KalVista Pharmaceuticals’ rare disease drug approval, coinciding with the agency's investigation of Sarepta's Duchenne muscular dystrophy therapy following patient deaths. These developments highlight ongoing regulatory challenges and scrutiny faced by novel therapies targeting rare and neuromuscular conditions, affecting biotech companies navigating approval pathways and safety evaluations. The saga underscores the complex dynamics between FDA leadership and pharmaceutical sponsors in managing innovation and patient safety.