Belief BioMed and Takeda China Announce Approval of China's First Hemophilia B Gene Therapy

In a significant development for the treatment of hemophilia B, Belief BioMed (BBM) and Takeda China have announced that their gene therapy product, BBM-H901 (Dalnacogene Ponparvovec Injection), has received approval from the National Medical Products Administration (NMPA) in China. This milestone marks BBM-H901 as the first gene therapy for hemophilia B to be approved in the country. BBM developed and manufactured the drug, while Takeda China is tasked with its commercialization across mainland China, Hong Kong, and Macau. The collaboration between these two companies aims to leverage their combined resources to make this groundbreaking therapy available to patients, potentially transforming the treatment landscape for hemophilia B.

Dr. Xiao Xiao, Co-founder and Chief Science Officer of BBM, emphasized the company's dedication to advancing gene therapy innovations. He noted that the approval of BBM-H901 represents a critical milestone for BBM, offering a novel treatment option for patients with hemophilia B. The therapy is anticipated to enable patients to lead more normal lives, and BBM plans to work closely with Takeda China to expedite its implementation and consider overseas commercialization to reach a broader patient population.

Sean Shan, Senior Vice President of Takeda Pharmaceutical and President of Takeda China, expressed honor in collaborating with BBM to supply China's first locally developed hemophilia B gene therapy. This approval complements Takeda's portfolio in the areas of hemophilia and rare diseases, and positions the company to provide innovative treatments to more patients. Takeda China intends to continue its strategic partnerships to accelerate the introduction of breakthrough therapies, thereby contributing to the advancement of China's rare disease sector.

Hemophilia B is a genetic disorder characterized by a deficiency of coagulation factor IX, which leads to excessive bleeding. Traditional treatments involve frequent infusions of prothrombin complex concentrates or factor IX, which can be burdensome. Repeated bleeding episodes can cause joint damage and disability, imposing significant physical and financial burdens on patients and their families.

BBM-H901 utilizes a recombinant adeno-associated virus (rAAV) vector to deliver an optimized human coagulation factor IX gene into liver cells, enabling continuous expression and secretion of the factor into the bloodstream. This process facilitates proper blood clotting and holds the potential to significantly improve patients' quality of life.

Professor Zhang Lei from the Blood Disease Hospital of the Chinese Academy of Medical Sciences, who led the clinical study on BBM-H901, highlighted the transformative potential of this therapy. Unlike traditional methods requiring frequent injections, BBM-H901 offers a one-time dosing that reduces bleeding and joint damage risks, thereby helping patients resume normal daily activities.

The investigational journey of BBM-H901 began in 2019 with the initiation of its first clinical trial. Subsequent research findings were published in prominent journals such as The Lancet-Hematology and The New England Journal of Medicine. These studies demonstrated promising results, with patients showing sustained factor IX activity without severe adverse events.

Following the Investigational New Drug (IND) application approval in China in 2021, BBM-H901 received Breakthrough Therapy Designation from China's Center for Drug Evaluation and Orphan Drug Designation from the U.S. FDA. In 2024, the therapy also received Rare Pediatric Disease Designation from the FDA and Advanced Therapy Medical Products status from the European Medicines Agency.

The latest Phase III clinical study findings, presented at the American Society of Hematology's 66th Annual Meeting, revealed a significant decrease in the annualized bleeding rate and a drastic reduction in the need for factor IX infusions post-gene therapy, with most participants experiencing no bleeding events. The ongoing follow-up aims to affirm long-term efficacy and safety.

In summary, the approval of BBM-H901 marks a new era in hemophilia B treatment in China, offering patients and their families hope for a less burdensome and more effective therapy. The collaboration between BBM and Takeda China exemplifies a strategic partnership poised to deliver innovative solutions to unmet medical needs.