The U.S. FDA has issued Capricor Therapeutics a complete response letter regarding its BLA for deramiocel, the company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
In the CRL, the FDA said that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and cited the need for additional clinical data. The CRL also referenced certain outstanding items in the chemistry, manufacturing and controls section of the application, but, according to a press release from Capricor, the company believes it has addressed these issues in prior communications with the FDA. Given the timing of the CRL issuance, Capricor says these materials were not reviewed by the FDA.
“We are surprised by this decision by the FDA. We have followed their guidance throughout the process. Prior to the CRL, the review had advanced without major issues, including a successful pre-licensure inspection and completion of the mid-cycle review,” said Linda Marbán, Ph.D., CEO of Capricor.
The agency offered Capricor the opportunity to request a Type A meeting to discuss the path forward. Capricor said it plans to engage further with the FDA to determine the appropriate next steps. The company will also submit data from the phase 3 HOPE-3 trial to provide additional evidence of effectiveness from an adequate and well-controlled study. The trial is a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected in the third quarter of 2025.
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.
Capricor’s BLA for deramiocel was granted priority review in March 2025 and had a PDUFA date of August 31, 2025.
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