PPMD is excited to learn that Capricor Therapeutics has completed the submission of its rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel (CAP-1002) for the treatment of Duchenne muscular dystrophy cardiomyopathy. Deramiocel is Capricor’s novel cell therapy thought to decrease inflammation and fibrosis while exerting positive effects on muscle regeneration.

Capricor announced its plans to file a BLA by year-end in September 2024 and has indicated that the BLA submission is supported by the company’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials compared to natural history data from an FDA funded and published dataset on the implications of Duchenne cardiomyopathy and potential biomarkers of disease progression. According to Capricor, the company has requested a priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority six-month review from the date the submission is accepted by the FDA.

PPMD is pleased to learn this news from Capricor and we look forward to further updates on deramiocel’s progress.

Read Capricor’s press release here.