Deramiocel (CAP-1002)
Duchenne muscular dystrophy · Patients with Duchenne muscular dystrophy and impaired skeletal muscle function
CAPR · Capricor Therapeutics
Full timeline
4 milestonesFDA decision expected after review extension
FDA extended the PDUFA target action date for the deramiocel BLA from August 22 to November 22, 2026 after accepting a major amendment containing HOPE-3 open-label-extension data and additional analyses.
Capricor — Deramiocel PDUFA extension↗FDA advisory committee reviewed the BLA
FDA convened the Cellular, Tissue and Gene Therapies Advisory Committee to review deramiocel. The committee's vote on substantial evidence for the originally proposed cardiomyopathy indication was 3 in favor and 9 against.
Capricor — Second-quarter regulatory update↗FDA resumed review of the Class 2 BLA resubmission
FDA accepted Capricor's response to the prior Complete Response Letter as a Class 2 resubmission and set the original August 22, 2026 action date.
Capricor — BLA review resumed↗HOPE-3 reported positive pivotal results
Deramiocel met the Phase 3 HOPE-3 primary endpoint for upper-limb function in Duchenne muscular dystrophy.
Capricor — Positive HOPE-3 topline results↗Related News
Capricor Therapeutics to Report Third Quarter 2026 Financial Results and Provide Recent Corporate Update on November 10
Oct 5, 2026Capricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data on Upper Limb Function in Duchenne Muscular Dystrophy at 2026 World Muscle Society Congress
Sep 17, 2026Capricor Therapeutics to Present HOPE-3 and HOPE-3 Open-Label Extension Data at 2026 World Muscle Society Congress
Aug 24, 2026Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA
Aug 13, 2026Capricor Therapeutics Reports Second Quarter 2026 Financial Results and Provides Corporate Update