Originally AUG 22 2026; extended to NOV 22 2026
(Following the July 2026 CTGTAC advisory committee meeting (9-3 against efficacy), Capricor submitted an amendment on 2026-08-24 including 24-month HOPE-3 open-label extension data and additional robustness analyses to support a refined proposed indication focused on upper-limb function (the HOPE-3 primary endpoint). CBER accepted the amendment for review, classified it as a Major Amendment, and extended the PDUFA by three months., announced AUG 24 2026).
About
Duchenne muscular dystrophy is a genetic disorder that progressively weakens all the body's muscles, including the heart, leading to life-threatening heart failure. Deramiocel is an investigational cell therapy made from donor-derived heart cells. When infused into a patient, these cells may help calm the damaging inflammation and scarring in the heart muscle, potentially slowing the decline in heart function.
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 month...
→AUG 31 2025 shifted to AUG 22 2026 (Original BLA received CRL Jul 2025 after AdComm 5-12 against efficacy. Class 2 resubmission accepted Q1 2026; new PDUFA established Mar 10 2026 with no identified review issues.; announced MAR 10 2026)
→AUG 22 2026 shifted to NOV 22 2026 (Following the July 2026 CTGTAC advisory committee meeting (9-3 against efficacy), Capricor submitted an amendment on 2026-08-24 including 24-month HOPE-3 open-label extension data and additional robustness analyses to support a refined proposed indication focused on upper-limb function (the HOPE-3 primary endpoint). CBER accepted the amendment for review, classified it as a Major Amendment, and extended the PDUFA by three months.; announced AUG 24 2026)
