FDA Extends PDUFA Review for Capricor’s Deramiocel

A late-stage data submission has reset Capricor Therapeutics' regulatory clock for Deramiocel, its investigational cell therapy for Duchenne muscular dystrophy, pushing the PDUFA target action date from August 22 to November 22, 2026. The three-month extension follows FDA's classification of Capricor's BLA amendment as a major amendment under 21 CFR Part 601 review procedures, a designation that carries direct timeline consequences regulatory affairs teams should account for in their own BLA planning.

The amendment, submitted in the wake of a July 2026 Advisory Committee meeting, packages 24-month open-label extension data from the pivotal Phase 3 HOPE-3 study alongside additional robustness analyses. Capricor simultaneously requested a refined proposed indication narrowed to upper limb function, the primary endpoint HOPE-3 met with statistical significance. FDA's Center for Biologics Evaluation and Research (CBER) accepted the amendment, citing significant unmet medical need in DMD, a population of approximately 15,000 patients in the United States with no curative options.

For regulatory affairs leads managing cell and gene therapy BLAs, the sequence here is instructive. Submitting supplemental Phase 3 data post-Advisory Committee, while potentially strengthening the evidentiary package, triggers CBER's major amendment classification criteria and automatically extends the review window. Teams navigating similar RMAT-designated programs should model this scenario into their submission timelines, particularly where open-label extension data is still maturing at the time of original BLA filing.

Deramiocel carries Orphan Drug, RMAT, and Rare Pediatric Disease designations in the U.S., and Orphan Drug and ATMP designations in Europe. The allogeneic cardiosphere-derived cell (CDC) therapy operates through an exosome-mediated mechanism, modulating macrophage phenotype to reduce fibrosis and preserve skeletal and cardiac muscle function. The RMAT designation affords Capricor enhanced FDA engagement, which the company characterised as constructive through the Advisory Committee process.

The November 22, 2026 action date now represents the measurable checkpoint against which CBER's review of the refined upper limb function indication will conclude.

Source: Capricor Therapeutics, Inc. via GlobeNewswire, August 24, 2026.