The FDA advisory committee voted against recommending approval of Capricor Therapeutics’ deramiocel, an allogeneic cardiosphere-derived cell therapy for Duchenne muscular dystrophy (DMD). The committee’s decision was based on the available data, which did not provide substantial evidence of effectiveness for deramiocel in treating cardiomyopathy in patients with DMD.
The discussion centered heavily on the phase 3 HOPE-3 trial findings, including the interpretation of its cardiac-function findings, missing data, and the statistical methods used to analyze the results. Linda Marban, CEO of Capricor, expressed concerns about the FDA’s briefing document, stating that parts of it were hard to reconcile with what actually occurred.
A major point of disagreement involved the statistical analysis plan. FDA reviewers based their efficacy assessment on an earlier version, while Capricor argued that a later version was the appropriate basis for interpreting the results. They extended their concerns beyond the dispute, questioning the magnitude and consistency of the observed cardiac effects.
Janet Turk Wittes, a member of the committee, explained that she voted no because the results were “fragile” and didn’t show enough evidence of benefit. Some panelists viewed the evidence for upper-limb function more favorably than the cardiac findings, but that discussion did not result in a separate affirmative approval recommendation.
The advisory committee’s recommendation is nonbinding, and the FDA is not required to follow it. The agency’s Prescription Drug User Fee Act target action date for the deramiocel application is August 22, 2026. Following the vote, analysts lowered their expectations for approval, with Piper Sandler expecting the FDA to issue another complete response letter rather than approve deramiocel for DMD cardiomyopathy.
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This is the second FDA review cycle for deramiocel. In July 2025, the FDA issued a complete response letter stating that the application did not provide substantial evidence of effectiveness. Capricor subsequently submitted additional clinical data from the phase 3 HOPE-3 trial, which met its primary end point, Performance of the Upper Limb version 2.0, as well as the key secondary cardiac end point of left ventricular ejection fraction.
DMD-associated cardiomyopathy is a major contributor to morbidity and mortality in Duchenne. Despite this, there is currently no FDA-approved therapy specifically indicated for DMD cardiomyopathy. If approved, deramiocel would introduce a cell-based treatment option into the DMD care pathway and could raise considerations around care access and the evidence needed to assess long-term clinical and economic outcomes.
For now, the FDA’s final decision on deramiocel remains pending, and the company will have to wait until August 22, 2026, to learn the outcome. The decision will have significant implications for patients with DMD and their families, who are waiting for effective treatments for this devastating disease.
The FDA’s evaluation of deramiocel is a complex process that involves careful consideration of the available data and the potential benefits and risks of the therapy. It’s possible that additional data or information will be needed before a final decision can be made.
Patients and families affected by DMD will continue to wait for news about this promising therapy.
