Priority Review for DMD Cardiomyopathy BLA

FDA has accepted for priority review an NS Pharma BLA for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. DMD primarily affects males and leads to progressive muscle weakness, loss of mobility, and eventually life-threatening cardiac and respiratory complications. The agency has set a user fee review action target date of 8/31.

Deramiocel comprises allogeneic cardiosphere-derived cells (CDCs), a type of stromal cell that has shown promising immunomodulatory, antifibrotic, and regenerative effects in preclinical and clinical studies, according to the company. These cells work by secreting extracellular vesicles known as exosomes, which can reprogram macrophages to adopt a healing rather than a pro-inflammatory response.

Deramiocel, which was developed by Capricor Therapeutics, is part of a distribution agreement that has NS Pharma responsible for commercialization and distribution of the therapy in the U.S.