Capricor’s Deramiocel BLA Under FDA Review with August 2026 PDUFA Date, Hits PUL v2.0 and LVEF Endpoints
Capricor’s BLA for Deramiocel in Duchenne muscular dystrophy is under FDA review with an August 22, 2026 PDUFA date. In the HOPE-3 Phase 3 trial, Deramiocel met its PUL v2.0 primary endpoint (p=0.03), key LVEF secondary endpoint (p=0.04) and showed 3.3% LVEF improvement plus 83% slowed functional decline.
1. BLA Under FDA Review
Capricor’s Biologics License Application for Deramiocel is under Class 2 resubmission review by the FDA with a PDUFA target action date of August 22, 2026, following a complete response letter resolution.
2. Positive Phase 3 HOPE-3 Results
The HOPE-3 trial enrolled 106 Duchenne muscular dystrophy patients and met the co-primary PUL v2.0 endpoint (p=0.03) and key LVEF secondary endpoint (p=0.04). Late-breaking analyses demonstrated a 3.3 percentage-point LVEF improvement (p=0.017) and an 83% slowdown in disease progression on the “eat 10 bites” task (p=0.018).
3. Commercial Readiness and Financials
Capricor’s San Diego GMP facility completed an FDA pre-license inspection with all Form 483 observations addressed and is operational for commercial launch. The company holds approximately $318 million in cash, funding operations through 2027 and supporting its transition to a commercial-stage biotech firm.