The National Medical Products Administration (NMPA) has officially approved BBM-H901 (Dalnacogene Ponparvovec Injection) for the treatment of adult patients with moderate to severe hemophilia B, marking a significant breakthrough in gene therapy for this rare bleeding disorder in China. Developed by Belief BioMed (BBM) and to be commercialized by Takeda China, this innovative treatment represents the first approved hemophilia B gene therapy in the country.
Transformative Treatment for Hemophilia B Patients
Hemophilia B, an inherited bleeding disorder caused by factor IX (FIX) deficiency, affects approximately 1 in 30,000 male births globally. Until now, Chinese patients have relied primarily on prothrombin complex concentrate (PCC) or FIX replacement therapy, requiring frequent intravenous injections that create substantial physical, emotional, and financial burdens.
BBM-H901 works through a fundamentally different mechanism than traditional treatments. The therapy utilizes a recombinant adeno-associated virus (rAAV) vector to deliver an optimized human coagulation FIX gene directly into patients' liver cells. These cells then continuously express and secrete coagulation FIX into the bloodstream, promoting normal clotting function with just a single administration.
Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of BBM, emphasized the significance of this approval: "The approval of BBM-H901 is an important milestone in the development of BBM. 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."
Impressive Clinical Results
The Phase III clinical trial results, presented at the 66th Annual Meeting of the American Society of Hematology (ASH) in December 2024, demonstrated remarkable efficacy. After 52 weeks of follow-up:
- Mean annualized bleeding rate (ABR) was reduced to just 0.6
- Average FIX activity reached 55.08 IU/dL (one Stage Assay SynthaSIL aPTT reagent)
- Average number of FIX infusions decreased from 58.2 times/year to only 2.9 times/year
- 80.8% of participants (21 of 26) experienced no bleeding events after treatment
- No serious adverse events were reported
Professor Zhang Lei from the Blood Disease Hospital of the Chinese Academy of Medical Sciences, who led the registered clinical study, described the approval as bringing "revolutionary hope" to Chinese hemophilia B patients. "By a single dosing, the risk of bleeding and joint damage can be reduced, and patients are back to a normal life," he stated.
Earlier investigator-initiated trials also showed promising results. A study published in The Lancet-Hematology and The New England Journal of Medicine in 2022 demonstrated that ten adult patients with moderate to severe hemophilia B achieved a mean FIX activity of 36.9 IU/dL after a median follow-up of 58 weeks, with no serious adverse events reported. Long-term follow-up data presented at the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress confirmed that FIX activity remained stable over three years, with no drug-related serious adverse events, thrombotic events, or inhibitors observed.
Strategic Partnership for Commercialization
The collaboration between Belief BioMed and Takeda China aims to accelerate patient access to this breakthrough therapy. BBM will handle development and manufacturing, while Takeda China will manage commercialization across mainland China, Hong Kong, and Macau.
Sean Shan, Senior Vice President of Takeda Pharmaceutical and President of Takeda China, highlighted the strategic importance of this partnership: "The approval of BBM-H901 will further enrich our portfolio in hemophilia and rare disease areas, and will help more patients to benefit from innovative treatments. In the future, Takeda China will continue to deepen our strategic cooperation with local partners, to accelerate the commercialization of more breakthrough therapies."
Regulatory Recognition and Future Prospects
BBM-H901 has received significant regulatory recognition beyond China. The therapy has obtained Breakthrough Therapy Designation from China's Center for Drug Evaluation, Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), Rare Pediatric Disease Designation from the FDA, and Advanced Therapy Medical Products designation from the European Medicines Agency (EMA).
These designations reflect the potential global impact of this therapy and may accelerate its availability to hemophilia B patients worldwide. BBM has expressed intentions to pursue overseas commercialization to benefit more patients globally.
Advancing Gene Therapy in China
This approval represents a milestone not only for hemophilia B treatment but also for China's growing gene therapy sector. As one of the first domestically developed gene therapies to receive regulatory approval in China, BBM-H901 demonstrates the country's increasing capabilities in advanced biopharmaceutical research and development.
For patients with hemophilia B, the approval offers hope for a dramatically improved quality of life with reduced bleeding episodes, fewer hospital visits, and potentially normal daily activities without the constant fear of bleeding events that can cause long-term joint damage.
The continuous follow-up of clinical trial participants will provide additional insights into the long-term safety and efficacy of this groundbreaking treatment, potentially establishing a new standard of care for hemophilia B management in China and beyond.
