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 (CAP-1002) for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
The committee concluded that the available clinical evidence does not provide substantial proof of deramiocel's effectiveness, dealing a severe blow to the investigational cell therapy's regulatory prospects.
A Contentious and Perplexing Meeting
The advisory committee meeting was highly contentious, characterized by intense disagreements between Capricor and FDA reviewers over which statistical analysis plan (SAP) should govern the pivotal trial results.12 Capricor's CEO, Linda Marbán, expressed profound frustration during the company's presentation, arguing that the FDA's briefing documents focused on a draft SAP that was never intended to support the final marketing application.
Despite Marbán's objections, committee members were ultimately unpersuaded by the clinical efficacy data, particularly regarding the Phase 3 HOPE-3 trial's secondary endpoints of cardiac function. Panel members described the data as "very fragile." Dr. Janet Turk Wittes, an expert in randomized controlled trials and a member of the committee, remarked:
"The word of the day seems to be fragile. The results were very fragile... I simply didn’t see enough evidence of benefit to vote yes."
Severe Unmet Need and Regulatory Outlook
The negative vote was particularly challenging for committee members given the devastating nature of DMD-associated cardiomyopathy—the leading cause of death in DMD patients—and the emotional testimonies delivered by families during the public hearing. While some committee members expressed deep sympathy, they felt bound by the lack of robust, statistically sound efficacy data.
Deramiocel, which consists of allogeneic cardiosphere-derived cells (CDCs) designed to exert immunomodulatory and anti-fibrotic effects, previously received a Complete Response Letter (CRL) in July 2025. While the FDA is not legally bound to follow the recommendations of its advisory panels, it typically aligns with them, meaning Capricor faces an uphill battle to secure standard approval in this cycle.
-
An instance of Post-unblinding changes to a statistical analysis plan doom even highly promising therapeutic signals. — Capricor's regulatory campaign faltered as the advisory committee rejected efficacy data tied to post-hoc, post-unblinding statistical modifications. ↩︎
-
An instance of Post-hoc statistical adjustments and single-arm designs cannot substitute for randomized clinical evidence. — The clinical efficacy data was deemed too fragile because the trial did not show a clear benefit under its original prespecified statistical parameters. ↩︎