Deramiocel PDUFA extended to November 22, 2026 with refined upper-limb DMD indication
- Sponsor
- Capricor Therapeutics $CAPR
- Drug
- deramiocel
- Indication
- Duchenne muscular dystrophy (DMD) cardiomyopathy; proposed refined indication: upper-limb function
FDA extended the PDUFA for Capricor's deramiocel BLA from August 22 to November 22, 2026 after Capricor submitted a Major Amendment adding 24-month HOPE-3 open-label extension data and requesting a refined indication focused on upper-limb function.
What this means
Deramiocel is a cell therapy Capricor is developing for boys with Duchenne muscular dystrophy, a fatal genetic muscle-wasting disease. Its FDA advisory committee voted 9-3 against approval on July 29 based on the original cardiomyopathy indication. Capricor has now submitted an amendment with 24-month follow-up data from its Phase 3 HOPE-3 trial and asked the FDA to consider a narrower indication focused on preserving upper-limb function, which was the trial's primary endpoint. The FDA accepted the amendment as a Major Amendment and pushed the target action date back by three months, from August 22 to November 22, 2026, to review the additional data.