Belief BioMed, a portfolio company of Qiming Venture Partners, announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially approved by China's National Medical Products Administration (NMPA) for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency). BBM-H901 is the first approved hemophilia B gene therapy in China, and is also the first approved gene therapy in China based on recombinant adeno-associated virus (rAAV) vector.
Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said: "Belief BioMed has always been committed to R&D and innovation in the field of cutting-edge gene therapy, and is dedicated to fulfilling the unmet medical needs. The approval of BBM-H901 is an important milestone along the development journey of Belief BioMed. As the first hemophilia B gene therapy drug independently developed in China, BBM-H901 will provide a new treatment option for hemophilia B patients, which is expected to provide patients with an innovative treatment solution and help them return to a normal life. In the future, Belief BioMed will strive to accelerate the overseas commercialization process of BBM-H901, hoping to benefit more hemophilia B patients."
Dr. Jane Zheng, Co-founder and Chief Executive Officer of Belief BioMed, said: "The market approval of BBM-H901 takes less than seven years from R&D to launch, which is a remarkable rapid speed. This is not only generated from the dedication and unremitting efforts of our employees, but also from the strong supports given by our external partners. The approval of BBM-H901, the first approved recombinant adeno-associated virus (rAAV) gene therapy in China, marks a new era of China's gene therapy industry and highlights Belief BioMed's advantages in the field. Relying on the advanced AAV capsid discovery platform Capsidx®, hundreds of key vector technologies, as well as an independent production platform, Belief BioMed has established an extensive R&D pipeline, including the pipelines on rare genetic diseases such as hemophilia and Duchenne muscular dystrophy, and those on chronic diseases such as osteoarthritis, Parkinson's disease and cervical intraepithelial neoplasia. All of those are developed to fulfill the high levels of unmet medical needs. In the future, Belief BioMed would continue to focus on gene therapy to bring cutting-edge technology to more patients and achieve our vision of serving patients worldwide."
BBM-H901, based on a recombinant adeno-associated virus (rAAV) vector, can deliver the optimized human coagulation FIX gene into liver cells of patients. Then, coagulation FIX is continuously expressed and secreted into the bloodstream using the host cell gene transcription system, thereby promoting coagulation.
In 2022, the research results of first Investigator-initiated Trial (IIT) of BBM-H901 were successively published on international authoritative journals, The Lancet-Hematology and The New England Journal of Medicine. In 2024, the IIT long-term follow-up results of more than 3 years were orally presented as an oral presentation at the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress, and in the same year, Belief BioMed released the results of the Phase 3 clinical study at the 66th Annual Meeting of the American Society of Hematology (ASH).
The current results showed that compared to treatment efficacy of preventive factor IX (FIX) replacement therapy, the annualized bleeding rate (ABR) of the patient was significantly reduced, and the FIX activity was rapidly increased and maintained stable expression, while showing good safety.
As an example, the results of BBM-H901 Phase 3 clinical study showed that after 52 weeks of follow-up, the mean annualized bleeding rate (ABR) of the participants was 0.6, the average FIX activity reached 55.08 IU/dL (one Stage Assay SynthaSIL aPTT reagent), and the average number of infusions of FIX drug decreased from 58.2 times/year before gene therapy treatment to 2.9 times/year after that. Twenty-one of the 26 participants (80.8%) had no bleeding events after treatment, and all participants had no SAEs.
In August 2022, BBM-H901 obtained the Breakthrough Therapy Designation by the Center for Drug Evaluation, China's National Medical Products Administration, and later on Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). In 2024, BBM-H901 obtained the Rare Pediatric Disease Designation (RPDD) from the FDA and the Advanced Therapy Medical Products (ATMP) from the European Medicines Agency (EMA).
Belief BioMed signed an exclusive cooperation agreement with Takeda China. According to the agreement, Belief BioMed granted Takeda China the exclusive license for the commercialization of BBM-H901 in Chinese Mainland, Hong Kong, and Macau.
