Capricor Therapeutics' Deramiocel Gains Orphan Drug and ATMP Status from EMA

On November 20, 2024, Capricor Therapeutics (NASDAQ: CAPR), a biotech firm focused on cell and exosome-based treatments for rare diseases, announced that the European Medicines Agency (EMA) has awarded both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to its principal therapeutic candidate, deramiocel, intended to treat Duchenne muscular dystrophy (DMD).

The Orphan Drug designation affords Capricor multiple benefits to aid the development of deramiocel in Europe, such as market exclusivity for a decade upon approval and significantly reduced regulatory fees. The ATMP designation offers extensive regulatory support to facilitate the development of cell-based therapies. These benefits can potentially shorten the time to market, streamline developmental processes, and provide access to critical resources, proving to be highly beneficial for companies creating innovative treatments.

Linda Marbán, Ph.D., the CEO of Capricor, stated, “Securing both the Orphan Drug and ATMP designations from the EMA marks considerable progress in our mission to deliver deramiocel to DMD patients globally. Along with the Orphan Drug and RMAT designations from the U.S. FDA, we have a chance to achieve market exclusivity for deramiocel in two major markets. These accomplishments underscore our dedication to developing novel, transformative therapies for those affected by DMD.”

Capricor recently initiated the rolling submission of its Biologics License Application (BLA) with the U.S. FDA, aiming for full approval of deramiocel to treat all DMD-related cardiomyopathy cases. The company plans to complete its full BLA submission by the end of 2024.

Deramiocel, also known as CAP-1002, comprises allogeneic cardiosphere-derived cells (CDCs), a group of stromal cells proven in preclinical and clinical studies to possess strong immunomodulatory, antifibrotic, and regenerative properties in treating dystrophinopathy and heart failure. CDCs function by secreting exosomes, which target macrophages and modify their expression profile to promote healing instead of inflammation. Over 100 peer-reviewed scientific publications have examined CDCs, and they have been administered to more than 200 human subjects in several clinical trials. The U.S. FDA has already granted deramiocel Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations for DMD treatment. Additionally, if approved, Capricor could receive a Priority Review Voucher (PRV), thanks to a previous rare pediatric disease designation.

Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle weakness and chronic inflammation affecting the skeletal, heart, and respiratory muscles, with a median mortality age of around 30 years. DMD affects approximately one in every 3,500 male births, with an estimated 15,000-20,000 patients in the United States. The disease is caused by defective production of functional dystrophin, a protein essential for muscle integrity. The lack of functional dystrophin leads to substantial muscle cell damage and death, eventually causing fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced by scar tissue, leading to cardiomyopathy and eventually heart failure, which is the primary cause of death in DMD. Currently, treatment options are limited and there is no cure.

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company committed to developing transformative cell and exosome-based therapies to revolutionize the treatment of rare diseases. Its lead product, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy, has shown promising immunomodulatory, antifibrotic, and regenerative effects in treating dystrophinopathies and heart disease. Deramiocel is currently in Phase 3 clinical development for DMD treatment. Additionally, Capricor is leveraging its exosome technology via its proprietary StealthX™ platform for preclinical development in various areas, including vaccinology and targeted delivery of therapeutics for a wide range of diseases. Capricor remains dedicated to exploring new possibilities and developing groundbreaking treatments for those in need.