Capricor Therapeutics (CAPR) saw its stock soar 5.04% on Wednesday, following news 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 in Europe, including 10 years of market exclusivity if deramiocel is approved, as well as reduced regulatory fees. The ATMP designation offers regulatory support to assist in the development of cell-based therapies like deramiocel, potentially reducing time to market and streamlining the development process.
Capricor's CEO Linda Marban expressed excitement about these designations, stating they are significant steps forward in bringing deramiocel to DMD patients worldwide. The company also announced it has initiated a rolling Biologics License Application (BLA) with the U.S. FDA and expects to complete the submission by the end of 2024, seeking full approval for deramiocel to treat DMD-cardiomyopathy.
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