Capricor Therapeutics has announced that the European Medicines Agency (EMA) has granted both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD). This decision marks a significant regulatory milestone for the development of this innovative cell-based therapy in Europe.
The Orphan Drug designation provides Capricor with market exclusivity for 10 years upon approval, along with reduced regulatory fees. The ATMP designation offers substantial regulatory support to streamline development and facilitate access to critical resources, potentially accelerating the path to market.
Significance of EMA Designations
According to Linda Marbán, Ph.D., Chief Executive Officer of Capricor, "Receiving the Orphan Drug and ATMP designations from the EMA are significant steps forward as we work to bring deramiocel to DMD patients worldwide." These designations, combined with Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. FDA, position Capricor to secure market exclusivity in key global markets.
Deramiocel: A Novel Therapeutic Approach
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), which have demonstrated potent immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies of dystrophinopathy and heart failure. These cells act by secreting extracellular vesicles, or exosomes, which modulate macrophage activity, shifting them from a pro-inflammatory to a healing phenotype. To date, deramiocel has been administered to over 200 human subjects across multiple clinical trials and is supported by over 100 peer-reviewed scientific publications.
Addressing Unmet Needs in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe genetic disorder affecting approximately one in every 3,500 male births. It is characterized by progressive muscle weakness, chronic inflammation, and eventual heart and respiratory muscle failure, with a median survival age of around 30 years. The underlying cause is a deficiency in functional dystrophin, a structural protein essential for muscle integrity. The lack of dystrophin leads to muscle cell damage, fibrosis, and ultimately, cell death. Cardiomyopathy, a common complication in DMD, often leads to heart failure and is a leading cause of mortality in these patients. Current treatment options are limited, highlighting the urgent need for new therapies.
Regulatory Pathway and Future Prospects
Capricor has initiated a rolling Biologics License Application (BLA) submission with the U.S. FDA, seeking full approval of deramiocel for treating all patients diagnosed with DMD-cardiomyopathy, with completion expected by the end of 2024. If approved, Capricor may also be eligible for a Priority Review Voucher (PRV) based on its rare pediatric disease designation.
