Business
Capricor Therapeutics Announces Intent to File Biologics License Application for Full Approval of Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy
-BLA to be Supported by Existing and Natural History Cardiac Data as Discussed with the FDA- -Initial Label Would Include All Patients with Cardiomyopathy
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"-BLA to be Supported by Existing and Natural History Cardiac Data as Discussed with the FDA- -Initial Label Would Include All Patients with Cardiomyopathy Associated with Duchenne Muscular Dystrophy- -Rolling Submission Planned to Commence in October 2024- -Internal GMP Manufacturing Established to Support BLA and Commercialization- -Investor Webcast Today at 8:30 a.m. ET- SAN DIEGO, Sept. 24, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today, following recent meetings with the U.S. Food and Drug Administration (FDA), its intent to file a Biologics License Application (BLA) based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. Following the FDA meetings: Capricor plans to commence the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024.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.In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024. “There are currently no approved therapies for DMD cardiomyopathy, which is the leading cause of death in those with Duchenne. Based on the strength of our cardiac data, combined with the FDA’s commitment to advancing therapeutics for the treatment of rare diseases, we are seeking approval for the cardiomyopathy associated with DMD and will look to expand the label for skeletal muscle myopathy post-approval,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “This approach is the result of multiple in-depth meetings with FDA where we showed robust and positive cardiac data from our HOPE-2 and HOPE-2 OLE studies compared to natural history data from a large cohort of patients.” Dr. ...