FDA pushes back Capricor's Deramiocel PDUFA date to November 22, 2026

Capricor Therapeutics said on August 24, 2026 that the FDA extended the PDUFA target action date for the Deramiocel biologics license application from August 22, 2026 to November 22, 2026. The extension follows Capricor's submission of additional Phase 3 HOPE-3 data and analyses supporting a refined proposed indication.¹

Following the July 2026 Advisory Committee meeting, Capricor added an amendment containing 24-month open-label extension results from the HOPE-3 trial along with further robustness analyses, asking the FDA to weigh both the existing and new data toward a narrower indication centered on upper limb function, which was HOPE-3's primary endpoint.¹ The FDA's Center for Biologics Evaluation and Research agreed to accept the amendment for review, pointing to the significant unmet medical need in DMD.¹ Because the agency classified the filing as a major amendment, the review period was extended by three months.¹

CEO Linda Marbán said the additional year of follow-up gives the company one of the most extensive datasets on upper limb function in Duchenne, and that HOPE-3 met its primary endpoint with a statistically significant benefit¹, according to the release.

Deramiocel is an investigational allogeneic cell therapy. It carries Orphan Drug, RMAT and Rare Pediatric Disease designations in the United States, plus Orphan Drug and ATMP status in Europe, and the Rare Pediatric Disease designation could make Capricor eligible for a Priority Review Voucher if the drug is approved.¹ DMD affects roughly 15,000 people in the United States.¹