for Duchenne muscular dystrophy cardiomyopathy
Originally AUG 31 2025; extended 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).
Duchenne muscular dystrophy is a genetic disorder that causes progressive muscle weakness, which often leads to serious heart complications as the disease advances. Deramiocel is an investigational cell therapy made from donor-derived heart cells. By delivering these cells directly into the bloodstream, the treatment may help reduce inflammation and scarring in the heart muscle of affected individuals.
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...
Source: ClinicalTrials.gov