FDA Advisory Committee Votes Against Efficacy of Capricor's Duchenne Cell Therapy Deramiocel
On July 29, 2026, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9–3 that the available clinical evidence does not support the effectiveness of Capricor Therapeutics’ lead cell therapy, deramiocel (CAP-1002), for treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). While the advisory panel's recommendation is nonbinding, the decisive negative vote introduces substantial regulatory headwinds for the biologics license application (BLA) ahead of its scheduled August 22, 2026, PDUFA target action date.
The contentious day-long meeting was dominated by a technical dispute over the appropriate Statistical Analysis Plan (SAP) for interpreting the pivotal Phase 3 HOPE-3 trial.1 FDA scientists raised serious concerns that Capricor altered key analyses after the study was unblinded. Specifically, the FDA argued that under the originally agreed-upon SAP version 1.0, the trial did not demonstrate a statistically significant benefit.2 Capricor strongly countered that SAP version 3.0, which they claim was finalized prior to unblinding, is the correct framework to assess the results. Although some committee members were encouraged by the therapy's secondary functional signal on upper limb skeletal muscle function (as measured by the PUL 2.0 scale), the majority concluded that the cardiac efficacy data were too fragile and plagued by study design and statistical discrepancies to support approval for DMD-associated cardiomyopathy.
"The Cellular, Tissue, and Gene Therapies Advisory Committee voted to reject the therapy 9 votes to 3. That vote is nonbinding, but it strongly signals a difficult path ahead for the company and deramiocel before the FDA’s August 22 decision date on whether to approve the therapy." — Inside Precision Medicine
"Several committee members who voted no said that discrepancies between the FDA's and Capricor's analyses of HOPE-3, together with inconclusive secondary cardiac findings, did not provide sufficient evidence of a clinically meaningful benefit." — Medscape
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An instance of Post-unblinding changes to a statistical analysis plan doom even highly promising therapeutic signals. — The advisory panel voted down the candidate because of disputes regarding post-unblinding statistical modifications. ↩︎
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An instance of Post-hoc statistical adjustments and single-arm designs cannot substitute for randomized clinical evidence. — Regulators rejected the drug candidate because the trial failed to show effectiveness under the original prespecified plan before post-unblinding adjustments were made. ↩︎