San Diego-based Capricor Therapeutics (Nasdaq: CAPR) announced that the US FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) will meet on July 29, 2026 to review the Biologics License Application (BLA) for deramiocel (CAP-1002), an allogeneic cell therapy for Duchenne muscular dystrophy (DMD). The AdCom meeting precedes a PDUFA target action date of August 22, 2026, placing a potential deramiocel FDA approval decision approximately eight weeks away.
The US FDA has scheduled the CTGTAC meeting as part of its ongoing BLA review for deramiocel in DMD. The AdCom convening is a procedural step in which the committee will assess the benefit-risk profile of the therapy and issue a non-binding recommendation. The BLA review is classified as a Class 2 resubmission — the US FDA having lifted a prior Complete Response Letter and resumed review in March 2026. The application is supported by positive Phase III HOPE-3 data demonstrating statistically significant improvement in upper limb function and cardiac measures.
Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that secrete extracellular vesicles (exosomes), which reprogramme macrophages from a pro-inflammatory to a pro-healing phenotype, exerting immunomodulatory and anti-fibrotic effects on both cardiac and skeletal muscle.
DMD is a rare progressive neuromuscular disorder with no curative therapy. Deramiocel's dual skeletal and cardiac muscle benefit profile is positioned as potentially first-in-class; existing approved therapies in DMD — including exon-skipping agents — address the underlying genetic defect but do not specifically target the cardiac deterioration that is the leading cause of death in the disease.