Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD). Capricor?s goal for this meeting will be to finalize its BLA filing plans based on all currently available data as well as to work with the FDA to outline the rolling BLA submission timeline. Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years.

It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000 to 20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement.

Treatment options are limited and there is no cure.