Takeda China and Conviction Pharmaceuticals Group (hereinafter referred to as Conviction Pharmaceuticals) announced today that the National Drug Administration (NDA) has formally accepted the New Drug Application (NDA) for BBM-H901 Injection for the treatment of adult patients with Hemophilia B. BBM-H901 Injection is a recombinant adeno-associated virus (AAV) gene therapy product that was independently developed and manufactured by Conviction Pharmaceuticals, and the commercialization of this product will be undertaken by Takeda China in the future. Takeda China is responsible for the commercialization of the product in Mainland China, Hong Kong, China and Macau, China. This milestone development means that the product is expected to bring new treatment options for hemophilia B patients in China.

Zhang Lei, the lead investigator of the registered clinical study of BBM-H901 injection and a professor at the Hospital for Hematological Diseases of the Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences), said, "There is still an unmet medical need for the hemophilia B patient population in China, which is currently treated with Plasminogen Complex (PCC) and Coagulation Factor IX as an alternative therapy, which is associated with problems such as risk of infection and inconvenience of administration. There are problems such as infection risk and inconvenient drug administration. At the same time, joint bleeding and deformities are common in patients with moderate to severe disease due to inadequate or unstandardized treatment. data from the BBM-H901 injection study have confirmed its safety and efficacy1,2 , and we look forward to the launch of this product and the clinical benefits it will bring to patients! "

BBM-H901 Injection

BBM-H901 Injection is used to prevent bleeding in adult patients with hemophilia B by introducing the human coagulation factor IX gene into patients with hemophilia B for sustained expression by intravenous administration to increase and maintain coagulation factor levels over time. 2022, BBM-H901 Injection was added to the "Breakthrough Therapeutic Varieties" list by the Center for Drug Review of the State Administration of Pharmaceutical Affairs. list. The results of the investigator-initiated clinical study (IIT) have been published in the prestigious international journals The Lancet - Hematology and The New England Journal of Medicine, and the results of the 3+ year long term follow up were presented in an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) Congress in 20243.

This submission is based on a multicenter, single-arm, open, single-administration registry clinical study (CTR20212816) designed to evaluate the safety and efficacy of a single intravenous infusion of BBM-H901 Injection in hemophilia B patients ≥18 years of age with endogenous coagulation factor IX (FⅨ) activity ≤2 IU/dL (i.e., ≤2%). To date, all subjects in its Phase III confirmatory study phase have completed the 52-week post-administration visit.