by: Parent Project Muscular Dystrophy
Capricor Therapeutics Initiates Rolling Submission of Biologics License Application (BLA) for Deramiocel (CAP-1002) for the Treatment of Duchenne Cardiomyopathy
Capricor Therapeutics announced it has begun the rolling submission of its Biologics License Application (BLA) with the FDA seeking full approval for deramiocel (CAP-1002) for the treatment of Duchenne-cardiomyopathy. This submission process is expected to be completed by the end of 2024. Once the rolling BLA submission is completed, the FDA will notify the company when it is formally accepted for review.
Capricor recently shared that the BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center. This is the first time an approval is being sought for a Duchenne product based on cardiac outcomes rather than skeletal muscle function or a dystrophin surrogate.
Capricor has also previously indicated that it plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study in order to support potential label expansion to treat Duchenne skeletal muscle myopathy. Capricor does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024.
PPMD is excited to see Capricor reach this exciting milestone of initiating the BLA submission and we have requested a webinar with the company to discuss updates. We will share additional information as it becomes available.
