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 weakens muscles over time, including the heart, leading to severe cardiomyopathy and reduced life expectancy. Deramiocel is a cell therapy derived from donor heart tissue that aims to repair damaged heart muscle by releasing proteins that reduce inflammation and promote healing. By targeting the heart muscle directly, deramiocel could potentially slow the progression of cardiomyopathy in patients with Duchenne muscular dystrophy.
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