regulatory
confidence high
sentiment positive
materiality 0.85
FDA accepts Capricor's BLA for deramiocel for DMD cardiomyopathy with Priority Review, PDUFA Aug 31, 2025
CAPRICOR THERAPEUTICS, INC.
- FDA accepted BLA for deramiocel (cell therapy) to treat Duchenne muscular dystrophy cardiomyopathy.
- Priority Review granted; PDUFA target date August 31, 2025.
- FDA has not identified any potential review issues at this time.
- If approved, deramiocel would be first approved therapy for DMD cardiomyopathy.
- Supports Phase 2 HOPE-2 and OLE trials vs. natural history data from FDA-funded dataset.