FDA Grants Capricor a Three-Month PDUFA Extension for Deramiocel in Duchenne Muscular Dystrophy
On August 24, 2026, Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) target action date for its Biologics License Application (BLA) for deramiocel (CAP-1002) from August 22, 2026, to November 22, 2026.
The three-month extension gives Capricor additional breathing room to secure approval for its investigational cell therapy, which is designed to treat Duchenne muscular dystrophy (DMD). The delay was triggered by Capricor submitting a significant amendment to its application, which the FDA classified as a "major amendment," requiring additional time to review.
The HOPE-3 Pivot and Refined Indication
- Major Amendment Details: The amendment contains newly generated 24-month open-label extension (OLE) data from Capricor's Phase III HOPE-3 clinical trial (NCT05126758).
- Narrows Indication: Along with the new data, Capricor submitted a refined proposed indication that narrows the focus of deramiocel's therapeutic label specifically to the preservation of upper limb function in DMD patients.
- Regulatory Context: This pivot follows a complex regulatory history. The FDA previously issued a Complete Response Letter (CRL) in July 2025 citing efficacy and CMC deficiencies. After Capricor resubmitted with completed HOPE-3 data, a subsequent FDA advisory committee meeting voted 9-3 against recommending approval. The new 24-month OLE data and narrowed indication represent Capricor's latest effort to address the agency's and advisory committee's concerns.
The FDA's review of the amended BLA is now set to conclude by the new target action date of November 22, 2026.