Capricor Therapeutics (NASDAQ: CAPR) has recently announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Biologics License Application (BLA) seeking full approval for Deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne Muscular Dystrophy (DMD) cardiomyopathy. The FDA has granted the BLA priority review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.
If approved, Deramiocel would be the first therapy to treat DMD cardiomyopathy. The FDA has not identified any potential review issues with the BLA at this time. Capricor is optimistic about the potential approval and expects Deramiocel to be a lifelong treatment, administered quarterly, with the potential to be widely adopted across the DMD-cardiomyopathy treatment landscape.
The BLA submission is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, compared to natural history data from an FDA-funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression.
Deramiocel has shown attenuation of DMD-cardiomyopathy in multiple clinical trials. The FDA grants priority review to applications for medicines that, if approved, provide significant improvements in the safety or effectiveness of the treatment of a serious condition. Deramiocel for the treatment of DMD has received orphan drug designation from the FDA and European Medicines Agency (EMA).
The regulatory pathway for Deramiocel is supported by Regenerative Medicine Advanced Therapy designation in the U.S. and the Advanced Therapy Medicinal Product designation in the European region. If Capricor were to receive FDA marketing approval for Deramiocel regarding the treatment of DMD by September 30, 2026, the company would be eligible to receive a Priority Review Voucher based on its previous receipt of a rare pediatric disease designation.
DMD is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles with mortality at a median age of approximately 30 years. The patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle.
Capricor Therapeutics, Inc. is dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. Its lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy, is currently in late-stage development for the treatment of Duchenne Muscular Dystrophy. Additionally, the company is harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins, and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. Following these announcements, the company's shares moved 2.1%, and are now trading at a price of $13.96. For more information, read the company's full 8-K submission here.
