FDA questions Capricor Duchenne cell therapy data; Capricor fires back

The FDA raised significant statistical concerns in briefing documents released ahead of today's adcomm meeting on Capricor Therapeutics' resubmitted BLA for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy-associated cardiomyopathy.

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. The agency said multiple post-study changes to the statistical analysis plan — including revised endpoint definitions, analytical methods and handling of missing data — were implemented after the randomized trial concluded and without prior FDA review. Reviewers also said a higher incidence of hypersensitivity reactions in the treatment arm may have functionally unblinded the study, limiting confidence in the post hoc findings.

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. The CRL also referenced certain outstanding items in the CMCsection of the application — issues that Capricor claimed it had already addressed in prior communications with the FDA.

In December 2025, Capricor submitted its response to the CRL, incorporating new phase 3 trial data. The company also shared positive topline results from its pivotal phase 3 HOPE-3 trial claiming the trial “delivered strong and definitive evidence that Deramiocel can meaningfully improve the course of Duchenne muscular dystrophy, demonstrating statistically significant improvements in both skeletal and cardiac function.”

Capricor fired back to the briefing doc today, sharing that The Lancet — which the company refers to as “one of the most selective and highly regarded peer-reviewed journals in medicine” has 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.”

Access to the adcomm meeting is here.