Capricor’s Deramiocel BLA Under FDA Review Ahead of August 22 PDUFA Date
CAPR•Capricor’s Deramiocel BLA for Duchenne muscular dystrophy is under full FDA review with a PDUFA target of August 22, 2026, after the HOPE-3 Phase 3 trial met its primary and all controlled secondary endpoints. The company holds $279 million cash, its GMP plant is expanding, and it sued NS Pharma.
1. Regulatory Review and Clinical Data
Capricor’s Biologics License Application for Deramiocel targeting Duchenne muscular dystrophy is under full FDA review with a PDUFA target date of August 22, 2026. The HOPE-3 Phase 3 trial met its primary endpoint on upper-limb function and all Type I error-controlled secondary endpoints, demonstrating statistically significant improvements versus placebo.
2. Manufacturing and Commercial Preparations
The company’s GMP manufacturing facility in San Diego is fully operational following a successful FDA pre-license inspection, and a second-floor expansion is on track to scale annual capacity to 2,000–2,500 patients (roughly 10,000 doses). A chief commercial officer with direct DMD experience is set to join in the coming weeks to lead launch preparations.
3. Financial Position and Legal Action
Capricor reported approximately $279 million in cash, funding operations into Q4 2027 and supporting launch activities. It filed suit against Nippon Shinyaku and NS Pharma seeking rescission of its U.S. distribution agreement to remove pricing barriers and remains eligible for a transferable Priority Review Voucher upon approval.
