FDA Advisory Committee Votes Against Capricor's Deramiocel in DMD Cardiomyopathy, Triggering Major BLA Amendment and PDUFA Extension
In late July 2026, Capricor Therapeutics suffered a significant regulatory setback as the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9 to 3 against recommending the approval of deramiocel (CAP-1002), an investigational cardiosphere-derived cell therapy for Duchenne muscular dystrophy (DMD).
However, in a dramatic late-August development, the FDA granted a three-month extension to the Biologics License Application (BLA) review timeline, allowing Capricor to submit additional long-term data and narrow its proposed indication to salvage the program.
The Contentious July 2026 Advisory Committee Meeting
The CTGTAC's negative vote was driven by concerns over the trial design and efficacy endpoints of the Phase 3 HOPE-3 study (NCT05126758).
- Endpoint Disagreement: Capricor had previously amended the HOPE-3 trial protocol to make left ventricular ejection fraction (LVEF) the primary efficacy endpoint (targeting DMD cardiomyopathy) and performance of the upper limb (PUL v2.0) a secondary endpoint.
- Fragile Data: FDA reviewers argued that the therapy failed to demonstrate a statistically significant difference between deramiocel and placebo at 12 months on its prespecified primary and secondary endpoints. Committee members characterized the clinical efficacy data as "very fragile," leading to the 9-3 vote against recommending approval.
Major BLA Amendment Resurrects the Program (August 24, 2026)
Following the negative advisory committee vote, Capricor engaged in intensive discussions with the FDA. Rather than waiting for a formal Complete Response Letter (CRL), Capricor submitted a major amendment to its BLA.
The amendment incorporates:
- 24-Month Open-Label Extension Data: Long-term, 24-month follow-up data from the Phase 3 HOPE-3 study.
- Refined Proposed Indication: A request that the FDA review the existing and new data in support of a narrower, refined indication focused specifically on the preservation of upper limb function (which was the original primary endpoint of HOPE-3 and demonstrated a statistically significant benefit) rather than broader cardiac function.
- Robustness Analyses: Additional statistical analyses of the existing data package to address the FDA's concerns.
New PDUFA Target Action Date: November 22, 2026
On August 24, 2026, Capricor announced that the FDA's Center for Biologics Evaluation and Research (CBER) accepted the amendment. Because the submission contains substantial new clinical data, the FDA classified it as a "major amendment."
Consequently, the FDA extended the Prescription Drug User Fee Act (PDUFA) target action date by three months, moving it from August 22, 2026, to November 22, 2026. The agency cited the substantial unmet medical need in the DMD population as a key factor in its willingness to review the new data and continue the BLA review process.