Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA

Core Viewpoint - Capricor Therapeutics has received FDA approval to resume the review of its Biologics License Application for Deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD), with a target action date set for August 22, 2026 [1][2][8] Company Overview - Capricor Therapeutics is a biotechnology company focused on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead product candidate [9] - The company aims to redefine treatment options for DMD, which affects approximately 15,000 individuals in the U.S., primarily boys [4][9] Product Details - Deramiocel (CAP-1002) is an allogeneic cardiac-derived cell therapy that has shown immunomodulatory and anti-fibrotic effects in preserving muscle function in DMD [5][9] - The therapy has received multiple designations, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation, which may qualify the company for a Priority Review Voucher upon approval [6][8] Clinical Development - The FDA's resumption of the BLA review follows the submission of data from the HOPE-3 clinical trial, which demonstrated positive results, including achievement of primary and secondary endpoints [2][8] - The company has administered Deramiocel to over 250 human subjects across multiple clinical trials, supported by extensive preclinical and clinical data [5][9] Regulatory Milestones - The FDA issued a Complete Response Letter in July 2025, which has now been lifted, allowing the review process to continue without identified issues [2][8] - The Prescription Drug User Fee Act (PDUFA) target action date for the BLA is set for August 22, 2026 [1][2][8] Commercialization Strategy - Capricor has entered into an agreement with Nippon Shinyaku Co., Ltd. for the exclusive commercialization and distribution of Deramiocel in the U.S. and Japan, pending regulatory approval [11]