FDA Advisory Committee Votes Against Capricor's Deramiocel in DMD Cardiomyopathy
In a major setback for Capricor Therapeutics, the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9 to 3 on July 29, 2026, against recommending the approval of deramiocel (human allogeneic cardiosphere-derived cells) for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
The negative vote capped off a highly contentious and chaotic session dominated by deep disagreements between Capricor and FDA reviewers over which statistical analysis plan (SAP) should govern the interpretation of the Phase 3 HOPE-3 (NCT00468923) trial results.1
Competing Statistical Analysis Plans and "Fragile" Data
The primary point of discord centered on competing versions of the trial's SAP:
- The FDA's Position: Reviewers evaluated efficacy based on an earlier, prespecified SAP version that they argued was the original methodology used to design and power the study. Under this analysis, the trial failed to demonstrate a robust statistical benefit.2
- Capricor's Position: The company asserted that SAP version 3.0, which was finalized and signed off on prior to unblinding, should control the interpretation of the study. Capricor maintained that under this plan, HOPE-3 met its primary endpoint of Performance of the Upper Limb version 2.0 (PUL 2.0) and its key secondary cardiac endpoint of left ventricular ejection fraction (LVEF).
The committee members ultimately sided with the FDA's cautious stance. Panelists raised significant concerns regarding missing data, analytical sensitivity, and the overall clinical and statistical consistency of the cardiac outcomes. Several experts characterized the efficacy findings as highly fragile and insufficient to prove reproducible clinical benefit.
Regulatory Context and Outlook
DMD-associated cardiomyopathy is a progressive, fatal condition and a leading cause of death in Duchenne patients, for which there are currently no FDA-approved therapies specifically indicated.
This represents the second regulatory cycle for deramiocel. In July 2025, the FDA issued a Complete Response Letter (CRL) citing inadequate evidence of effectiveness and outstanding chemistry, manufacturing, and controls (CMC) issues. Capricor subsequently resubmitted its BLA with Phase 3 HOPE-3 data, which the FDA accepted, establishing a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026.
Following the 9-3 advisory rejection, market expectations have shifted dramatically. Wall Street analysts (including Piper Sandler) have downgraded Capricor and lowered their expectations, anticipating that the FDA will issue a second CRL rather than grant approval, despite the nonbinding nature of the committee's vote.
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An instance of Post-unblinding changes to a statistical analysis plan doom even highly promising therapeutic signals. — Capricor's BLA faces rejection because the developer modified the trial's statistical analysis plan prior to unblinding, creating a major dispute with the FDA over the data's validity. ↩︎
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An instance of Post-hoc statistical adjustments and single-arm designs cannot substitute for randomized clinical evidence. — The FDA and its advisory committee rejected the therapy's efficacy claims because they relied on late-stage changes to the statistical plan rather than the original prespecified design. ↩︎