Capricor, backed by phase 3 data, plans to submit CRL response to FDA

Capricor Therapeutics says it's ready to submit its response to the CRL received from the FDA for its investigational cell therapy for the treatment of cardiomyopathy associated Duchenne muscular dystrophy, incorporating new phase 3 trial data.

The company shared positive topline results from its pivotal phase 3 HOPE-3 trial evaluating deramiocel, demonstrating statistically and clinically significant treatment effects on both upper limb function and cardiomyopathy.

The company was hit with a surprise CRL 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 chemistry, manufacturing and controls section of the application.

After a Type A meeting with the FDA, company shared a regulatory update in September, saying that the CMC items identified in the CRL had been addressed and that the HOPE-3 trial should serve as the “additional study” requested in the CRL.

Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in dystrophiopathies, such as DMD.