San Diego – October 19, 2026 -- The U.S. Food and Drug Administration has pushed the decision date for Capricor Therapeutics' Deramiocel Biologics License Application by three months, from August 22, 2026 to November 22, 2026, after classifying the company's latest data submission as a major amendment.
Deramiocel is an investigational cell therapy for Duchenne muscular dystrophy (DMD). Capricor (NASDAQ: CAPR) disclosed the extension of the Prescription Drug User Fee Act (PDUFA) target action date on the day of release.
Capricor files 24-month HOPE-3 extension data after July advisory committee
Following the July 2026 Advisory Committee meeting, Capricor submitted a BLA amendment containing 24-month open-label extension data from its pivotal Phase 3 HOPE-3 study. The filing also includes additional robustness analyses.
The company asked the FDA to review both the existing and new data in support of a refined proposed indication focused on upper limb function, the primary endpoint of HOPE-3.
CBER accepts amendment, citing unmet need in DMD
The FDA's Center for Biologics Evaluation and Research (CBER) accepted the amendment for review, citing the significant unmet medical need in DMD. The agency extended the target action date by three months to allow additional review time.
Capricor CEO Linda Marbán said HOPE-3 met its primary endpoint, with a statistically significant benefit in upper limb function. She said the added year of follow-up gives the company one of the most extensive clinical datasets on upper limb function in Duchenne.
Regulatory designations could unlock a priority review voucher
Deramiocel holds Orphan Drug, RMAT and Rare Pediatric Disease designations in the U.S., and Orphan Drug and ATMP designations in Europe. The Rare Pediatric Disease designation may qualify Capricor for a Priority Review Voucher upon approval.
DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Deramiocel has not been approved for commercial use in any indication.