FDA CRL - Capricor, Inc.

On July 9, 2025, the U.S. Food & Drug Administration issued a Complete Response Letter (CRL) to Capricor, Inc. for its Biologics License Application for deramiocel. The FDA concluded that the application could not be approved due to several deficiencies, primarily concerning clinical efficacy data and, implicitly, issues related to Chemistry, Manufacturing, and Controls (CMC), though specific CMC details were redacted.

The main clinical issue was that the HOPE-2 study failed to meet its prespecified primary and secondary efficacy endpoints for neuromuscular function. While Capricor submitted post hoc analyses from the HOPE-2 and HOPE-2-OLE studies to support deramiocel's effectiveness for cardiomyopathy in Duchenne muscular dystrophy (DMD) patients, the FDA found these insufficient. The agency noted that the HOPE-2 study was not originally designed to assess cardiomyopathy and lacked adequate prespecified objectives and multiplicity adjustments for its numerous exploratory cardiac endpoints, rendering these findings exploratory and potentially unreliable.

Operating under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act, which mandate substantial evidence of effectiveness from adequate and well-controlled studies, the FDA requires Capricor to conduct new studies. These studies must be adequate and well-controlled, with the primary objective of evaluating cardiac outcomes in deramiocel-treated DMD cardiomyopathy patients compared to a randomized, concurrent control group. Capricor must resubmit or withdraw its application within one year.