Recent regulatory disruptions at the FDA have surfaced amid scrutiny of Duchenne muscular dystrophy (DMD) gene therapy approvals. Sarepta Therapeutics experienced sudden FDA holds and shipment pauses for Elevidys, with communication lapses causing distress for patients and clinicians. Reports highlight FDA information leaks to media before notifying companies, intensifying uncertainty. Further turmoil involved Capricor Therapeutics’ advisory committee cancellation and agency leadership conflicts. The FDA’s cancer drug review chief, Vinayak Prasad, played a pivotal role in denying Replimune’s melanoma therapy RP1, reflecting deeper agency dysfunction and leadership changes impacting cell and gene therapy approvals.