FDA Extends PDUFA Date for Capricor’s DMD Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has extended the target action date for its decision on whether to approve Capricor Therapeutics’ Deramiocel by three months, to November 22, 2026.

The extension is intended to give regulators more time to review newly submitted clinical data. It follows Capricor’s submission of additional Phase 3 HOPE-3 data supporting a narrower proposed indication centered on upper limb function.

Duchenne muscular dystrophy is a rare, progressive genetic disorder that primarily affects boys. While advances in skeletal muscle therapies have emerged, there are currently no approved treatments specifically targeting the cardiomyopathy that ultimately drives mortality in the disease.

Deramiocel (CAP-1002) is an allogeneic cell therapy derived from cardiosphere-derived cells, designed to exert immunomodulatory and anti-fibrotic effects through exosome signaling. The therapy has been studied in more than 250 patients and has received multiple regulatory designations, including Orphan Drug, RMAT, and Rare Pediatric Disease designations in the U.S.

The delay comes after a contentious July 2026 FDA Advisory Committee meeting, where panelists voted 9-3 against the effectiveness of Deramiocel for treating cardiomyopathy in Duchenne muscular dystrophy (DMD), with the FDA’s own briefing documents arguing the therapy failed to show a statistically significant benefit over placebo at 12 months.

In response, Capricor submitted an amendment incorporating 24-month open-label extension data from HOPE-3, along with additional robustness analyses, asking the FDA to instead evaluate the therapy for a narrower indication focused specifically on upper limb function, which was the trial’s primary endpoint.

The FDA’s Center for Biologics Evaluation and Research classified the submission as a major amendment, given the significant unmet need in DMD, and extended the review clock accordingly.

This is not Capricor’s first regulatory detour. The FDA previously issued a Complete Response Letter in July 2025 before lifting it and resuming review in March 2026 under a Class 2 resubmission, with the now-superseded PDUFA date of August 22.

Despite the adcomm setback on cardiomyopathy, the HOPE-3 trial did meet its prespecified primary endpoint on upper limb function, showing a 54 percent slowing of disease progression on the Performance of Upper Limb scale versus placebo, in a 106-patient randomized, placebo-controlled study.