Belief BioMed's Gene Therapy BBM-H901 Receives Approval in Macao for Hemophilia B Treatment
核心洞察
Belief BioMed (搜索)'s BBM-H901 (Dalnacogene Ponparvovec Injection (搜索)) has been approved by Macao's Pharmaceutical Administration Bureau for treating adult patients with moderate to severe hemophilia B (搜索).
The gene therapy demonstrated significant efficacy in Phase 3 trials with a mean annualized bleeding rate of only 0.6 and sustained factor IX levels of 55.1 IU/dL at 52 weeks.
BBM-H901 uses a single injection to enable sustained factor IX expression, potentially eliminating the need for lifelong frequent injections required by conventional therapies.
Belief BioMed (搜索) announced that BBM-H901 (Dalnacogene Ponparvovec Injection (搜索)) has received official approval from the Pharmaceutical Administration Bureau of Macao Special Administrative Region of China for treating adult patients with moderate to severe hemophilia B (搜索). This milestone follows the therapy's approval in mainland China in April 2025, establishing BBM-H901 as China's first gene therapy for hemophilia B in both regions.
The approval addresses a significant unmet medical need in Macao while advancing global accessibility of this breakthrough gene therapy. Takeda China (搜索) is responsible for commercializing the product across mainland China, Hong Kong, and Macao.
Revolutionary Gene Therapy Approach
BBM-H901 represents a paradigm shift in hemophilia B (搜索) treatment through its innovative gene therapy design. The therapy utilizes a recombinant adeno-associated virus (rAAV) vector to precisely deliver the high-activity factor IX gene into patients' liver cells, enabling sustained and high-level expression of human factor IX within the liver and its subsequent secretion into the bloodstream.
The therapy incorporates several advanced features: an AAV843 engineered liver-tropic capsid for targeted delivery, the FIX (搜索)-Padua variant as the functional transgene, and a robust liver-specific promoter developed by Belief BioMed (搜索). Additionally, the product adopts a double-stranded AAV genome design, which facilitates rapid transgene expression following intravenous administration.
Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed (搜索), emphasized the therapy's potential impact: "BBM-H901 is a gene therapy designed to enable sustained and stable expression of coagulation factor IX in the body through a single administration, potentially freeing patients from the physical, psychological, and economic burdens associated with lifelong frequent injections required by conventional therapies."
Clinical Trial Results Demonstrate Strong Efficacy
The Phase 3 clinical study was a multicenter, single-arm trial evaluating the efficacy and safety of a single BBM-H901 injection in 26 adult patients with moderate to severe hemophilia B (搜索). After 52 weeks of follow-up, results showed remarkable improvements in patient outcomes.
The mean annualized bleeding rate (ABR) in participants was only 0.6 (95% CI: 0.18-1.99), representing a significant decrease compared to the superiority margin of 5.0 ABR for Chinese hemophilia B (搜索) patients receiving prophylactic treatment. The mean factor IX activity level at week 52 after gene therapy reached 55.1 IU/dL (SD35.9).
Treatment burden was dramatically reduced, with the average number of coagulation factor IX drug infusions decreasing from 58.2 times per year before gene therapy to just 2.9 times per year after treatment. Joint bleeding frequency significantly decreased, resulting in the number of target joints being reduced to zero.
Long-term Safety and Durability
An earlier investigator-initiated trial provided valuable long-term data, enrolling 10 adult participants with moderate to severe hemophilia B (搜索) who received a single BBM-H901 injection. Five-year follow-up results demonstrated sustained therapeutic benefit, with 70% of participants maintaining factor IX activity levels greater than 35 IU/dL and 40% achieving levels above 50 IU/dL at the final visit (median time to last visit was 210 weeks, range: 159-270 weeks).
The therapy exhibited an excellent safety profile across clinical trials, with no grade 3-4 adverse events, serious adverse events, or inhibitors observed. The studies continue with ongoing follow-up to monitor long-term outcomes.
Addressing Critical Medical Need
Hemophilia B (搜索) is an inherited bleeding disorder caused by factor IX deficiency. Traditionally, patients have relied on prothrombin complex concentrate or coagulation factor IX drugs as replacement therapy. Persistent and frequent bleeding often leads to joint structure and function damage, resulting in high disability rates and bringing significant physical pain, severe daily life restrictions, infection risks, blood clot risks, and substantial economic burden to patients and families.
Regulatory Recognition and Global Expansion
BBM-H901 has received significant regulatory recognition worldwide. The therapy obtained Breakthrough Therapy Designation from China's National Medical Products Administration in 2022 and was approved by NMPA in mainland China in April 2025. Internationally, BBM-H901 received Orphan Drug Designation from the U.S. FDA in 2022, FDA Rare Pediatric Disease Designation and European Medicines Agency Advanced Therapy Medicinal Product classification in 2024, and Orphan Drug Designation from the Saudi Food and Drug Administration.
The clinical results have been published in prestigious journals including The Lancet-Hematology, The New England Journal of Medicine, and Nature Medicine, with findings presented at major conferences including the International Society on Thrombosis and Haemostasis Congresses and the American Society of Hematology Annual Meeting.