The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee has voted that available evidence does not support the effectiveness of Capricor Therapeutics’ Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). 

DMD is a rare and life-threatening disease for which no approved cardiomyopathy treatment currently exists. 

Deramiocel (CAP-1002) 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 muscular dystrophies such as DMD.  

CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. 

The company says the Committee’s voting question addressed a narrower indication than it had proposed and did not include a vote on Deramiocel’s overall benefit-risk profile.  

In a separate discussion on upper limb function, the Committee’s feedback was directionally supportive of the clinical evidence from the Phase III HOPE-3 trial, including results on its primary endpoint, PUL 2.0. 

“We remain committed to Deramiocel and to the patients who could benefit from it,” said Linda Marbán, Chief Executive Officer of Capricor. “The Advisory Committee gave us an important opportunity to present the clinical evidence, and we were encouraged by the Committee’s discussion of Deramiocel’s impact on skeletal muscle.  

“We remain confident in the strength of the HOPE-3 data. In a moving open public hearing, patients, families, and clinicians shared their experience with the therapy, underscoring the unmet need within the Duchenne community. We remain focused on working with the FDA toward potential approval ahead of our 22 August 2026 PDUFA target action date.”