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Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA
Deramiocel has the potential to become the first therapy to address both skeletal and cardiac manifestations of Duchenne muscular dystrophyBLA supported by
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"Deramiocel has the potential to become the first therapy to address both skeletal and cardiac manifestations of Duchenne muscular dystrophyBLA supported by positive pivotal HOPE-3 Phase 3 results, including achievement of the primary endpoint and all Type I error-controlled secondary endpointsPDUFA target action date set for August 22, 2026; Company expects to be eligible for a Priority Review Voucher upon potential approval SAN DIEGO, March 10, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (“FDA”) has lifted the previously issued Complete Response Letter and resumed review of its Biologics License Application (“BLA”) seeking full approval of Deramiocel, an investigational cell therapy, for the treatment of Duchenne muscular dystrophy (“DMD”) cardiomyopathy. The submission has been classified as a Class 2 resubmission, with a Prescription Drug User Fee Act (“PDUFA”) target action date of August 22, 2026. “We are encouraged by the FDA’s acknowledgment of our response to the Complete Response Letter and its continued review of our BLA for Deramiocel,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “We believe the positive HOPE-3 results and broader clinical evidence reinforce Deramiocel’s potential to become a first-in-class therapy for Duchenne muscular dystrophy, with the opportunity to address both skeletal and cardiac manifestations of the disease. We look forward to continuing to work closely with the FDA throughout the review process and remain focused on bringing this important therapy to patients as expeditiously as possible.” The Company received a Complete Response Letter (“CRL”) from the FDA in July 2025. Following submission of data and supporting documentation from the HOPE-3 clinical trial, the FDA resumed review of the application and assigned a PDUFA target action date of August 22, 2026. At this time, the FDA has not identified any potential review issues in its response to the Company. Capricor also expects to be eligible to receive a Priority Review Voucher (“PRV”) upon potential approval of Deramiocel. About Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic di...