July 29, 2026 —
A U.S. Food and Drug Administration advisory committee voted against the efficacy of deramiocel, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy-associated cardiomyopathy.
The panel voted 9 to 3 against deramiocel’s efficacy in cardiomyopathy, following a meeting focused on Capricor’s resubmitted Biologics License Application. Although advisory committee recommendations are not binding, the FDA will consider the vote as it reviews the therapy. The agency’s decision is expected by August 22.
Deramiocel consists of cardiosphere-derived cells sourced from donor cadavers and is administered by intravenous infusion every three months. The therapy is intended to restore or preserve cardiac and skeletal muscle function in patients with Duchenne muscular dystrophy, or DMD. Capricor’s current application, however, is specifically focused on DMD-associated cardiomyopathy.
The advisory committee discussion centered on data from the Phase 3 HOPE-3 trial, as well as earlier data from the HOPE-2 trial and its open-label extension. Capricor and the FDA disagreed on whether the HOPE-3 study met its endpoints, with much of the discussion focused on statistical analysis plans and whether changes made before unblinding were appropriate.
For the cardiomyopathy endpoint, the review focused on left ventricular ejection fraction, or LVEF, a measure of the heart’s ability to pump blood. Capricor shifted its analysis from change in LVEF to ranked change in LVEF shortly before data unblinding. Even under the company’s preferred analysis, the LVEF result presented to the committee had a p-value of 0.09, which was not statistically significant.
Several committee members described the data as fragile, meaning that the conclusions appeared highly dependent on the specific statistical method used and the exclusion or inclusion of certain patients. Some experts also raised concerns about data suggesting increased left ventricular volume in deramiocel-treated patients.
Although the committee was asked to vote only on deramiocel’s efficacy in DMD-associated cardiomyopathy, many patient advocates, clinicians, and public commenters emphasized the therapy’s potential effects on skeletal muscle function and patient independence. Capricor also urged the panel to consider the broader disease context.
The FDA and Capricor also disagreed on the interpretation of Performance of the Upper Limb 2.0, or PUL 2.0, data. Capricor argued that the HOPE-3 trial met the primary skeletal muscle endpoint using percent change from baseline, while the FDA maintained that the originally defined mean change from baseline analysis was not statistically significant.
Committee members acknowledged that the upper limb data may be worth further investigation but generally found the evidence insufficient to support efficacy in the current application. Several panelists encouraged Capricor to continue studying upper limb function in future research.
The negative advisory committee vote adds uncertainty to deramiocel’s regulatory path. The FDA may still choose to approve the therapy, but the panel’s concerns around statistical robustness, endpoint interpretation, and cardiomyopathy-specific efficacy will likely weigh heavily in the agency’s final decision.
