Capricor says FDA sets Nov. 22 PDUFA date for deramiocel in Duchenne muscular dystrophy
CAPR•HOPE-3 results and financial position
Phase 3 HOPE-3 met its primary endpoint at 12 months, showing a 4.55% Performance of Upper Limb benefit versus placebo (p=0.029).
Capricor said it had cash of USD 238 million, which it believes supports runway through 2027, excluding potential product revenue or monetization of a priority review voucher.
The company also flagged priority review voucher eligibility, with an estimated potential sale value of about USD 150 million to USD 200 million if awarded.
FDA sets review timeline for deramiocel
Capricor says the FDA has outlined a review timeline for deramiocel in Duchenne muscular dystrophy, with a PDUFA target date of Nov. 22, 2026.
The company said the target label focus has narrowed to upper-limb impairment in patients older than 9 years, aligning with the HOPE-3 primary endpoint.
