Capricor Secures European Medicines Agency Designations For Duchenne Therapy

Capricor Therapeutics, a biotech company focused on innovative cell and exosome-based treatments for rare diseases, announced that the European Medicines Agency (EMA) has awarded Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to its leading therapy, deramiocel, for treating Duchenne muscular dystrophy (DMD).

The Orphan Drug designation offers significant advantages, including a decade of market exclusivity in Europe followed by approval and reduced regulatory fees. Meanwhile, The ATMP designation offers significant regulatory assistance to aid in developing cell-based therapies. Additional support could potentially shorten the time to market, simplify development, and enable better access to critical resources, making it a precious tool for companies developing innovative therapies.

Linda Marbán, Ph.D., Chief Executive Officer of Capricor, said in a statement, “Receiving the Orphan Drug and ATMP designations from the EMA are significant steps forward as we work to bring deramiocel to DMD patients worldwide. With these designations from the EMA, along with Orphan Drug and RMAT designations from the U.S. Food and Drug Administration (FDA), we have the opportunity to secure market exclusivity for deramiocel in two of the world’s key markets. These milestones reflect our commitment to advancing novel, transformative treatments for patients suffering from DMD.”

In addition, Capricor recently began a rolling submission of its Biologics License Application (BLA) with the U.S. FDA, seeking full approval for deramiocel to treat all DMD-cardiomyopathy patients. The company anticipates finalizing the submission by the close of 2024.