Capricor will file this year for FDA approval of Deramiocel for the treatment of Duchenne cardiomyopathy

As an early investor in Capricor Therapeutics, CureDuchenne is happy to share that Capricor has announced their intent to apply to the FDA for full approval of Deramiocel (CAP-1002) for the treatment of Duchenne cardiomyopathy.  

This BLA (Biologics License Application) 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 sets, as well as conversations with the FDA.  Capricor is expected to complete the filing by year-end 2024.  If successful, this would be a first-in-class therapy for Duchenne cardiomyopathy. 

The initial label being pursued is for all patients with cardiomyopathy associated with Duchenne. Capricor will later seek to expand the label to also treat Duchenne skeletal muscle myopathy by combining Cohorts A and B of the Phase 3 HOPE-3 trial, to serve as a post-approval study.  Therefore, Capricor will not unblind Cohort A and report that topline data, as was anticipated to occur by the end of this year. 

Deramiocel (CAP-1002) consists of cardiosphere-derived cells (CDCs) that secrete extracellular vesicles known as exosomes that are thought to promote anti-inflammatory and antifibrotic actions of the immune system.  

Read the full press release here or below