EMA Grants Orphan Drug and ATMP Status to Capricor's Deramiocel for Duchenne Muscular Dystrophy
Key Insights
The European Medicines Agency (EMA) has granted Orphan Drug Designation to deramiocel for treating Duchenne muscular dystrophy (search) (DMD (search)), providing market exclusivity and reduced regulatory fees.
Deramiocel also received Advanced Therapy Medicinal Product (ATMP) designation from the EMA, streamlining development and offering regulatory support for this cell-based therapy.
These EMA designations, along with FDA Orphan Drug and RMAT designations, aim to secure market exclusivity for deramiocel in key global markets.
Capricor Therapeutics (NASDAQ: CAPR) has received Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations from the European Medicines Agency (EMA) for its lead asset, deramiocel, in the treatment of Duchenne muscular dystrophy (search) (DMD (search)). These designations are significant milestones in Capricor's efforts to bring deramiocel to DMD patients worldwide.
The Orphan Drug designation confers several benefits, including 10 years of market exclusivity upon approval and reduced regulatory fees, facilitating deramiocel's development in Europe. The ATMP designation provides substantial regulatory support, potentially reducing time to market and streamlining development of this cell-based therapy.
Significance of EMA Designations
"Receiving the Orphan Drug and ATMP designations from the EMA are significant steps forward as we work to bring deramiocel to DMD (search) patients worldwide," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. These designations, combined with Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration (FDA), offer the opportunity to secure market exclusivity in key global markets.
Capricor has recently initiated a rolling Biologics License Application (BLA) submission process with the U.S. FDA, seeking full approval of deramiocel for treating all patients diagnosed with DMD (search)-cardiomyopathy, with completion expected by the end of 2024.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), stromal cells that have demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies of dystrophinopathy and heart failure. CDCs secrete extracellular vesicles, known as exosomes, which modulate macrophage expression to promote a healing phenotype. Deramiocel has been administered to over 200 human subjects across multiple clinical trials and is supported by over 100 peer-reviewed scientific publications. Deramiocel has previously received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation (RMAT) from the U.S. FDA. If approved, Capricor is also eligible for a Priority Review Voucher (PRV) based on a previous rare pediatric disease designation.
Duchenne Muscular Dystrophy (DMD)
DMD (search) is a genetic disorder characterized by progressive weakness and chronic inflammation of skeletal, heart, and respiratory muscles, with a median mortality age of approximately 30 years. It affects approximately one in every 3,500 male births, with an estimated patient population of 15,000-20,000 in the United States. DMD is caused by impaired production of functional dystrophin (search), leading to muscle cell damage, death, and fibrotic replacement. Cardiomyopathy, resulting in heart failure, is the leading cause of death in DMD patients. Current treatment options are limited, and there is no cure.