Capricor Therapeutics has announced that its primary product candidate, deramiocel, has received Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations from the European Medicines Agency (EMA) for the treatment of Duchenne muscular dystrophy (DMD). These designations aim to expedite the development and potential market availability of deramiocel in Europe.
The Orphan Drug designation provides Capricor with a decade of market exclusivity upon approval and significantly reduces regulatory fees. The ATMP designation is expected to streamline the development process for this cell-based therapy, potentially accelerating its time to market and providing access to essential resources.
Significance of EMA Designations
Linda Marbán, Ph.D., CEO of Capricor, emphasized the importance of these designations in the global effort to provide deramiocel to DMD patients. These European designations complement the Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations previously granted by the U.S. Food and Drug Administration (FDA).
Capricor has initiated a rolling Biologics License Application (BLA) with the FDA, targeting full approval of deramiocel for DMD-cardiomyopathy, with expectations to complete the submission by year-end. The company's San Diego facility is production-ready, and plans for a new manufacturing site are underway to meet anticipated demand.
About Deramiocel (CAP-1002)
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells, which have demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies. These cells release exosomes that influence macrophages to promote healing. Over 200 patients have received CDCs in various trials, and deramiocel is currently in Phase 3 development for DMD treatment.
DMD is a severe genetic disorder characterized by progressive muscle weakness and chronic inflammation, with an average life expectancy of about 30 years. It affects roughly one in 3,500 male births, with an estimated 15,000-20,000 patients in the U.S. The disease results from a lack of functional dystrophin, a protein essential for muscle integrity, leading to cell damage and fibrosis.
Capricor's Broader Strategy
Capricor is also exploring opportunities to expand the treatment's use to Becker muscular dystrophy and advancing its StealthX exosome technology for future therapeutics. The company's partnership with Nippon Shinyaku Co., Ltd. for the commercialization of deramiocel in the U.S. and Japan is contingent on regulatory approvals. Deramiocel is not yet approved for any indications, and Capricor's exosome-based candidates have not received clinical investigation approval.
