FDA to hold adcomm meeting for Capricor Duchenne cell therapy

Capricor Therapeutics says that the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee is planning to convene a committee meeting to discuss the company's resubmitted BLA seeking approval of deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

The company 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 chemistry, manufacturing and controls section of the application — issues that Capricor claimed it had already addressed in prior communications with the FDA.

With the adcomm meeting scheduled for July 29 and a PDUFA date of August 22, Capricor CEO Linda Marbán, Ph.D., expressed confidence in the totality of evidence supporting deramiocel, telling BioSpace she was surprised by the need for the meeting.

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.