FDA adcomm votes against approval of Capricor cell therapy

The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted that available evidence did not support the effectiveness of Capricor Therapeutics’ deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy.

In a closely watched meeting, the committee voted 3 for and 9 against approval. The committee was asked to vote specifically on deramiocel’s effectiveness in cardiomyopathy. According to Capricor, the voting “did not include a vote on deramiocel's overall benefit-risk profile. In a separate discussion on upper limb function, the committee's feedback was directionally supportive of the clinical evidence from the phase 3 HOPE-3 trial, including results on its primary endpoint, PUL 2.0.”

Capricor was hit with a surprise CRL for deramiocel in July 2025 in which regulators said that the BLA did not meet the statutory requirement for substantial evidence of effectiveness, citing the need for additional clinical data. In December 2025, Capricor submitted its response to the CRL, incorporating new phase 3 data from the HOPE-3 trial, in which the company said deramiocel met trial endpoints.

The FDA, however, disagreed and prior to the adcomm meeting raised significant statistical concerns in briefing documents. In the 53-page briefing doc, the FDA said the HOPE-3 study failed to meet its pre-specified primary and secondary efficacy endpoints and questioned the validity of the sponsor's subsequent analyses. At the same time, Capricor revealed that The Lancet had published the results from the HOPE-3 clinical trial. According to Capricor, “The manuscript underwent independent expert peer review, providing external validation of the trial’s design, statistical methodology and findings.”

The PDUFA date for deramiocel, which consists of allogeneic cardiosphere-derived cells, is August 22, 2026.