Belief BioMed announced BBM-H901 (Dalnacogene Ponparvovec Injection), China's first hemophilia B gene therapy, was officially approved in Macao, China
SHANGHAI, March 19, 2026 /PRNewswire/ -- Belief BioMed ("BBM") today announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially approved by the Pharmaceutical Administration Bureau of Macao Special Administrative Region of China for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency). This product is developed and manufactured by BBM, and Takeda China is responsible for its commercialization in mainland China, Hong Kong, China and Macao, China. Following its approval in mainland China in April 2025 as the country's first gene therapy for hemophilia B, [1] the recent approval of BBM-H901 in Macao, China, not only addresses the previously unmet medical need for such treatment in the region but also marks a crucial step in accelerating the global availability of this breakthrough gene therapy.
Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of BBM, said: "As the first gene therapy drug for hemophilia B in both Mainland China and Macao, China, [1],[2] we are delighted to leverage Macao's position as an international gateway to extend the reach of this innovative achievement to the Macao region and even broader markets. This approval reflects the Macao Special Administrative Region Government's forward-looking support for cutting-edge medical technologies and provides patients in the region with a breakthrough treatment option that keeps pace with international standards. BBM-H901 is a gene therapy designed to enable sustained and stable expression of coagulation factor IX in the body through a single administration, [3] potentially freeing patients from the physical, psychological, and economic burdens associated with lifelong frequent injections required by conventional therapies. Looking ahead, BBM will continue to collaborate with Takeda China to accelerate the accessibility of innovative therapies, and hope this approval is an opportunity to deepen our integration with the international market and enable China's advanced gene therapy to benefit more patients worldwide."
Hemophilia B is an inherited bleeding disorder caused by the deficiency of factor IX (FIX). [4] For a long time, patients can only rely on prothrombin complex concentrate (PCC) or coagulation FIX drugs as a replacement therapy. [5],[6] Persistent and frequent bleeding frequently leads to damage of joint structure and function, resulting in a high disability rate. [7] This not only brings great physical pain and severe restrictions on normal daily lives to patients, but also comes with the risk of infection, blood clots, etc. [5],[6] The high medical treatment costs also bring a heavy and constant economic burden on the patient's family.
BBM-H901 is a gene therapy drug based on a recombinant adeno-associated virus (rAAV) vector. It can precisely deliver the high-activity FIX gene into the liver cells of patients, enabling the sustained and high-level expression of human FIX within the patients' livers and its subsequent secretion into the bloodstream, thereby exerting the procoagulant activity of human FIX. The capsid of BBM-H901 is AAV843, an engineered liver-tropic capsid for targeted delivery. The functional transgene of BBM-H901 is FIX-Padua variant, a mainstream therapeutic gene for hemophilia B gene therapy, and is driven by a robust liver-specific promoter developed by the BBM to enable high-level FIX expression specifically in the liver. Furthermore, the product adopts a double-stranded AAV (dsAAV) genome design, which facilitates rapid transgene expression following intravenous administration. [3]
In 2019, the first Investigator-initiated Trial (IIT) of BBM-H901 was launched, and in 2021, the pivotal clinical trials was accepted for initiation in China. The related research results were successively published on international authoritative journals, The Lancet-Hematology, [7] The New England Journal of Medicine [8] and The Nature Medicine. [9] The Phase 3 clinical study was a multicenter, single-arm trial designed to evaluate the efficacy and safety of a single injection of BBM-H901 in adult patients with moderate to severe hemophilia B, enrolling a total of 26 eligible participants. The results of Phase 3 showed that after 52 weeks follow-up, the mean annualized bleeding rate (ABR) in participants was only 0.6 (95% CI: 0.18-1.99), with a significant decrease compared to the superiority margin, an average ABR of 5.0 for Chinese hemophilia B patients receiving prophylactic treatment. The mean FIX:C level at week 52 after gene therapy was 55.1 IU/dL (SD35.9) (OSA, SynthAsil). The average number of infusions of coagulation FIX drug decreased from 58.2 times/year (SD 30.67) before gene therapy treatment to 2.9 times/year (SD 10.71) after that. The number of joint bleeding frequency significantly decreased with the result that the number of target joints was reduced to zero. [9] The earlier-initiated IIT was a single-center, single-arm study that enrolled 10 adult participants with moderate to severe hemophilia B. These patients received a single injection of BBM-H901 and were subsequently followed for long-term observation. Five-year follow-up results of the 10 participants showed that, as of the final visit [median time to the last visit was 210 weeks (range: 159-270 weeks)], 70% of the participants maintained FIX:C levels greater than 35 IU/dL, with 40% achieving FIX:C levels above 50 IU/dL (one Stage Assay actin FSL aPTT reagent). [9] In addition, BBM-H901 exhibited good safety in clinical trials, with no grade 3-4 adverse events (AEs), serious adverse events (SAEs), or inhibitors observed. [9] The studies are still in the process of continuous follow-up.
Due to BBM-H901's positive efficacy and safety data, the follow-up results of the IIT study were also presented as oral reports at the International Society on Thrombosis and Haemostasis (ISTH) Congresses in 2024 and 2025 [10] respectively. The results of the Phase 3 clinical study were released at the 66th Annual Meeting of the American Society of Hematology (ASH) in 2024. [11]
BBM-H901 obtained the Breakthrough Therapy Designation by the Center for Drug Evaluation, [12] the National Medical Products Administration (NMPA) in 2022, and was approved by NMPA in Mainland China in April 2025. Additionally, BBM-H901 was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) in 2022, received FDA Rare Pediatric Disease Designation (RPDD) as well as Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA) in 2024, and was granted ODD by the Saudi Food and Drug Administration (SFDA). [13]
References |
[1] Up to April 2025. https://www.nmpa.gov.cn/yaopin/index.html |
[2] Up to March 2026.https://www.isaf.gov.mo/ |
[3] https://www.beliefbiomed.com/newsd-825.html |
[4] Thrombosis and Hemostasis Group, Chinese Society of Hematology, Chinese Medical Association / Hemophilia Treatment Center Collaborative Network of China. Chinese guidelines on the treatment of hemophilia (version 2020). Chin J Hematol.2020;41(4):265-271 |
[5] Mortensen GL, et al. Haemophilia. 2018;862-872 |
[6] Srivastava A, et al. Haemophilia. 2020;001-158.P95 |
[7] Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513 |
[8] Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624 |
[9] Xue F, et al. Nat Med. 2025 Nov 20 |
[10] Mankai Ju, et al. 2025 ISTH. OC 69.2 |
[11] Feng X, et al. 2024 ASH. Poster 3582 |
[12] https://www.cde.org.cn/main/xxgk/listpage/da6efd086c099b7fc949121166f0130c |
[13] https://www.beliefbiomed.com/newsd-852.html |
About Belief BioMed
Belief BioMed Inc. (BBM) is a global biotech company that integrates the research and development, manufacturing and clinical application of gene therapy products. The company is committed to providing innovative and more effective gene therapies for severe genetic and chronic diseases through safe and efficient viral vector technology. BBM has developed hundreds of key vector technologies, including HEK293 cell suspension serum-free culture process and full-scale chromatography purification process, and has established a commercial production platform for gene therapy drugs. The company has been building up its capabilities in a variety of fields including novel AAV capsids targeting different tissues, efficient transgene expression cassette design, and advanced clinically applicable vector manufacturing process. It has also established an extensive R&D pipeline covering a wide range of unmet clinical needs in different therapeutic areas such as hemophilia, DMD, Parkinson's disease, osteoarthritis, etc. Several product pipelines have entered clinical studies or submitted IND filings. The Biologics License Application (BLA) of a gene therapy for the treatment of adult patients with hemophilia B, has been approved in Mainland China and Macao China.
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