FDA extends PDUFA date for Capricor Duchenne therapy

The U.S. FDA has extended the PDUFA target action date for Capricor Therapeutics’ BLA for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy, from August 22, 2026 to November 22, 2026.

In a closely watched adcomm meeting in July, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 9-3 that available evidence did not support the effectiveness of Capricor’s deramiocel for the treatment of cardiomyopathy in patients with DMD. A few weeks later, Capricor said it would amend its BLA for deramiocel, narrowing the application’s focus to upper-limb skeletal muscle function — the primary efficacy endpoint of the phase 3 HOPE-3 trial — rather than cardiomyopathy.

Capricor submitted an amendment to the BLA that includes 24-month open-label extension data from its pivotal phase 3 HOPE-3 study and additional robustness analyses. CBER accepted the amendment for review, citing the significant unmet medical need in DMD. The FDA has classified the submission as a major amendment and extended the PDUFA target action date by three months to allow additional time to review the information.

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.