NS Pharma Inc, a subsidiary of Japan-based Nippon Shinyaku Co Ltd (TYO:4516), announced on Monday that the US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) filing for deramiocel, an investigational cell therapy, for the treatment of patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.

The Prescription Drug User Fee Act (PDUFA) target action date for the BLA Priority Review is 31 August 2025. The FDA has not identified any potential review issues so far.

Nippon Shinyaku signed an exclusive distribution agreement with Capricor for deramiocel in the United States in January 2022. NS Pharma will manage commercialisation and distribution of deramiocel in the United States.

NS Pharma president Yukiteru Sugiyama, PhD, said: "Deramiocel has the potential to address a clear, unmet medical need for patients diagnosed with DMD. We are excited for the possibility to bring additional treatment options and renewed hope to families of the rare disease community."