Capricor Therapeutics Granted Orphan Drug and ATMP Status for Deramiocel by European Medicines Agency
Thursday, November 21, 2024
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today 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).
The Orphan Drug designation provides Capricor with several benefits that support the development of deramiocel in Europe, including market exclusivity for 10 years if approval is granted and substantially reduced regulatory fees. The ATMP designation provides substantial regulatory support to assist in the development of cell-based therapies. The additional support can potentially reduce time to market, streamline development, and open up access to critical resources, making it an invaluable tool for companies working on innovative therapies.
“Receiving the Orphan Drug and ATMP designations from the EMA are significant steps forward as we work to bring deramiocel to DMD patients worldwide,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “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.”
Recently, Capricor announced that it had initiated its rolling Biologics License Application (BLA) submission process with the U.S. FDA seeking full approval of deramiocel to treat all patients diagnosed with DMD-cardiomyopathy. Capricor expects to complete its full BLA submission by the end of 2024.
Source: globenewswire.com
