Deramiocel FDA Approval Status
Last updated by Judith Stewart, BPharm on March 10, 2025.
FDA Approved: No
Generic name: deramiocel
Company: Capricor Therapeutics
Treatment for: Duchenne Muscular Dystrophy
Deramiocel is an investigational cell therapy in development for the treatment of Duchenne muscular dystrophy cardiomyopathy.
- Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. It is caused by the impaired production of functional dystrophin (a structural protein in muscle) which leads to cell damage and ultimately muscle cell death and fibrotic replacement. The leading cause of death in DMD patients is heart failure due to cardiomyopathy, which occurs as heart muscle cells cells progressively die and are replaced with scar tissue.
- Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure.
- A Biologics License Application (BLA) has been submitted to the U.S. Food and Drug Administration (FDA) for deramiocel to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. The BLA was supported by cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials.
- On March 4, 2025, Capricor Therapeutics announced that the FDA had accepted for review its BLA seeking full approval for deramiocel, and had assigned a PDUFA target action date of August 31, 2025.
Development timeline for deramiocel
Further information
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