Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy

Core Insights - Capricor Therapeutics is progressing towards the FDA's PDUFA target action date of August 31, 2025, for its Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy [1][2] - The FDA's mid-cycle review meeting indicated no significant deficiencies in the BLA submission, and an advisory committee meeting will be scheduled [1][2] - DMD is a severe genetic disorder affecting approximately 15,000-20,000 patients in the U.S., characterized by progressive muscle weakness and heart failure [3] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead product candidate [7] - Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic effects in clinical studies [4][7] - The company has received Orphan Drug Designation from the FDA and EMA for deramiocel, along with RMAT and ATMP designations to support its regulatory pathway [5] Clinical Development - The BLA submission for deramiocel is backed by data from Phase 2 trials (HOPE-2 and HOPE-2 OLE) and comparisons to FDA-funded datasets on DMD-cardiomyopathy [2] - Efficacy data from the ongoing HOPE-3 study is not included in the current BLA submission [2] - Capricor has entered an agreement with Nippon Shinyaku Co., Ltd. for the exclusive commercialization of deramiocel in the U.S. and Japan, pending regulatory approval [9]