Deramiocel (CAP-1002) - (HOPE-3)

August 24, 2026: Capricor Therapeutics (NASDAQ: CAPR) said the FDA extended the PDUFA target action date for its BLA for Deramiocel in Duchenne muscular dystrophy from August 22, 2026 to November 22, 2026 after accepting a major amendment.

The amendment, submitted after the July 2026 Advisory Committee meeting, includes 24-month open-label extension data from the pivotal Phase 3 HOPE-3 study plus additional robustness analyses. Capricor said the submission is intended to support a refined proposed indication focused on upper limb function, the primary endpoint of HOPE-3.

According to the company, HOPE-3 met its primary endpoint, showing a statistically significant benefit in upper limb function. FDA’s Center for Biologics Evaluation and Research accepted the amendment for review and extended the review by three months to assess the new information, citing the significant unmet need in DMD.

Deramiocel remains investigational and has not been approved for commercial use. The next key catalyst is the revised FDA action date of November 22, 2026.