Takeda and Belief BioMed's Gene Therapy for Hemophilia B Gains Regulatory Approval in China

On Thursday, a significant announcement came from Takeda, a Japanese pharmaceutical company, in collaboration with the local biotech firm Belief BioMed, regarding their new gene therapy drug, Dalnacogene Ponparvovec Injection. The product has gained the nod from China's National Medical Products Administration to be used for treating adult patients suffering from moderate to severe hemophilia B, making it the first gene therapy approved for this condition within the country.

Belief BioMed was responsible for the development and production of this innovative injection, while Takeda China will oversee its commercialization efforts across mainland China, Hong Kong, and Macao. In this joint venture, both companies plan to utilize their respective expertise to not only make this therapy accessible to patients more swiftly but also to explore advanced treatment options for hemophilia B.

Sean Shan, who holds the position of senior vice-president at Takeda and leads the company's operations in China, expressed pride in collaborating with Belief BioMed to launch the first locally developed gene therapy for this disease. He indicated that this approval represents a pivotal expansion for Takeda's strategic framework and product offerings aimed at addressing rare diseases.

Looking ahead, Takeda China is poised to strengthen its partnerships with local firms to accelerate the research and commercialization of additional pioneering therapies, ultimately ensuring that innovations from China reach more patients and foster significant growth in the realm of rare disease treatment.

From the perspective of Xiao Xiao, co-founder and chief science officer of Belief BioMed, there is a clear objective to extend the commercialization of their groundbreaking research to international markets, thereby broadening the reach of this treatment for hemophilia B.

Traditionally, hemophilia B has been treated through the infusion of prothrombin complex concentrate or Factor IX, which necessitates regular intravenous injections for life, exposing patients to ongoing bleeding risks that can severely affect their joint health and overall disability levels. According to Professor Zhang Lei, who led the clinical trials leading to this drug's registration, the approval of this new therapy offers significant hope to patients in China.

Zhang emphasized that with just a single injection, patients could see a decrease in their risk of bleeding and joint complications, eliminating the burden of frequent intravenous treatments. He is optimistic that ongoing advancements in research and real-world experience will herald a new era in hemophilia treatment, ultimately improving patients' quality of life.