PPMD is excited to learn that the FDA has accepted Capricor Therapeutics’ Biologics License Application (BLA) for review, seeking traditional approval for deramiocel (CAP-1002). Additionally, the FDA granted the BLA Priority Review, reducing the review timeline from the standard 10-month to a priority six-month review from the date the submission is accepted by the FDA. Under the Prescription Drug User Fee Act (PDUFA), the FDA’s target action date is August 31, 2025 to complete its review and announce its decision.

Deramiocel is Capricor’s novel cell therapy for the treatment of individuals diagnosed with Duchenne cardiomyopathy, which is thought to decrease inflammation and fibrosis while exerting positive effects on muscle regeneration. Capricor reports that, if approved, it expects deramiocel to be a lifelong treatment, administered quarterly through intravenous (IV) infusion.

The BLA submission is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data on Duchenne cardiomyopathy and potential biomarkers of disease progression. 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. If approved, deramiocel would be the first Duchenne-specific therapy  for cardiomyopathy.

We look forward to additional updates from Capricor as the review process continues, and we are grateful to all of the families who have participated in these trials. We are hopeful that deramiocel will be another approved therapeutic option for the Duchenne community.

Read Capricor’s press release here.

UPCOMING WEBINAR

Capricor will join PPMD for a community webinar on Thursday, March 6, 2025 at 1:00 PM ET to discuss this update. Register for the webinar here and submit any questions in advance here.