Capricor Begins BLA Submission With U.S. FDA For Deramiocel A Treatment Of DMD

Capricor Therapeutics is a biotech company prominently focused on developing cell- and exosome-based therapies for rare diseases. It has begun the rolling submission of its Biologics License Application (BLA) to the U.S. FDA for full approval of deramiocel—a medication aimed to treat patients with Duchenne muscular dystrophy (DMD) cardiomyopathy. 

Linda Marbán, Ph.D., Chief Executive Officer of Capricor, said in a statement, “This announcement marks an essential step in the U.S. regulatory process towards a potential Biologics License Application approval of deramiocel for the treatment of DMD. “This announcement marks a vital step for the U.S. regulatory process towards a potential Biologics License Application approval of deramiocel for the treatment of DMD

 Capricor anticipates to complete the submission by the end of 2024. The BLA could qualify for priority review, as deramiocel offers potential improvements in the safety and effectiveness of treatment for DMD cardiomyopathy, a condition with no currently approved therapies. After submission, the FDA will notify Capricor when the application is accepted for review.