Belief BioMed Group (BBM), a Shanghai-based gene therapy specialist, and Takeda have announced that the National Medical Products Administration (NMPA) has accepted for review the New Drug Application (NDA) for BBM-H901, a gene therapy for hemophilia B. This development marks a significant milestone for the treatment, which is being designed as both a prophylactic and a treatment for bleeding episodes in adults with hemophilia B. BBM-H901 is noted as the first adeno-associated virus (AAV) gene therapy for hemophilia B to receive Investigational New Drug (IND) approval for a registrational clinical study in China.
In an agreement reached in October 2023, BBM licensed the commercial operation rights of the drug to the Japanese pharmaceutical company Takeda, covering mainland China, Hong Kong, Macau, and Taiwan. The filing for approval is supported by data from a multi-center, single-arm, open-label, single-administration regulatory study (CTR20212816), which evaluated the safety and efficacy of a single intravenous infusion of BBM-H901 in patients with hemophilia B who are aged 18 years or older and have endogenous coagulation factor IX (F IX) activity of 2 IU/dL or less, equivalent to 2% or less. All subjects in the Phase III confirmatory study phase have completed the 52-week visit following administration.- Flcube.com
