FDA Extends Capricor Deramiocel PDUFA Date

On August 24, 2026, Paragon Therapeutics announced that the U.S. United States — Food and Drug Administration (FDA) extended the PDUFA target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22, 2026 to November 22, 2026. The extension follows Capricor's submission of an amendment to the BLA, which includes 24-month open-label extension data from the pivotal Phase 3 HOPE-3 study and additional robustness analyses, supporting a refined proposed indication focused on upper limb function. The FDA's United States — Center for Drug Evaluation and Research (CBER) accepted the amendment for review, citing significant unmet medical need, and classified it as a major amendment requiring additional review time. HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function. Deramiocel holds Orphan Drug, RMAT, and Rare Pediatric Disease designations in the U.S., and Orphan Drug and ATMP designations in Europe. The company continues to work with the FDA as it completes its review.