FDA lifts CRL for Capricor Duchenne cell therapy

The FDA has lifted the previously issued complete response letter and resumed review of Capricor Therapeutics’ BLA seeking full approval of deramiocel, the company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

The submission has been classified as a Class 2 resubmission, with a PDUFA target action date of August 22, 2026. If approved, deramiocel has the potential to become the first therapy to address both skeletal and cardiac manifestations of Duchenne muscular dystrophy.

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 — issues that Capricor claimed it had already addressed in prior communications with the FDA.

After a Type A meeting with the FDA, company shared a regulatory update in September 2025, 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.