Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has extended the PDUFA target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD). The extension allows the FDA additional time to review a major amendment submitted by the company.

Key Details

  • New PDUFA Date: The target action date has been moved by three months, from August 22, 2026, to November 22, 2026.
  • Reason for Extension: The company submitted a major amendment to the BLA, which included 24-month open-label extension data from its pivotal Phase 3 HOPE-3 study.
  • Refined Indication: The amendment supports a refined proposed indication focused on upper limb function, which was the primary endpoint of the HOPE-3 study.