November 2026 preview: 4 FDA decisions to watch in pediatric care

Four rare disease therapies with stakes for children and adolescents face US Food and Drug Administration (FDA) decisions within 10 days of each other this month. Between November 17 and 27, the FDA is due to act on treatments for infantile-onset Niemann-Pick disease type C, Duchenne muscular dystrophy (DMD), the neurological manifestations of type 3 Gaucher disease, and limb-girdle muscular dystrophy type 2I/R9, according to their Prescription Drug User Fee Act (PDUFA) target action dates. The most contested is Capricor Therapeutics' deramiocel for DMD, which reaches its decision after a 2025 complete response letter and a July 2026 advisory committee meeting at which FDA reviewers disputed its phase 3 results.²,³