Capricor Therapeutics, Inc. (CAPR) a clinical-stage biotechnology company, on Wednesday, announced that the European Medicines Agency has granted Orphan Drug and Advanced therapy medicinal product (ATMP) designations for its lead asset, Deramiocel, for treating Duchenne muscular dystrophy (DMD).
DMD is a 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. Currently, around 15,000 to 20,000 DMD-affected people are reported in the United States.
Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic, and regenerative actions in dystrophinopathy and heart failure. Deramiocel is currently advancing through Phase 3 clinical development.
The drug previously received Orphan Drug and Regenerative medicine advanced therapy (RMAT) designations from the FDA.
The Orphan Drug and ATMP designations from the EMA for Deramiocel in Europe provide 10 years of market exclusivity upon approval, along with significantly reduced regulatory fees, support, and assistance in the development of cell-based therapies.
In the U.S., the company has initiated its rolling Biologics License Application submission seeking full FDA approval for Deramiocel in the treatment of patients with DMD-cardiomyopathy, based on Phase 2 HOPE-2 and HOPE-2 open-label extension (OLE) trials. The full submission is expected to be completed by the end of this year.
Also of note is the company's proprietary StealthX exosome-based vaccine for the prevention of SARS-CoV-2, which is currently in the manufacturing process, with plans for delivery to the National Institute of Allergy and Infectious Diseases (NIAID) in the first quarter of 2025.
CAPR closed Wednesday's trading at $18.92, up by 2.49 percent on the Nasdaq.
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