Belief BioMed, Takeda China win approval for China’s first hemophilia B gene therapy

Belief BioMed and Takeda China jointly announced that BBM-H901 has been approved by the National Medical Products Administration for the treatment of adult patients with moderate to severe hemophilia B, marking the first approved hemophilia B gene therapy in China.

Takeda China, through an exclusive licensing deal inked with Belief in 2023, will commercialize the asset in mainland China, Hong Kong China, and Macau China.

BBM-H901, based on a recombinant adeno-associated virus (rAAV) vector, delivers the optimized human coagulation FIX gene into liver cells of patients. Coagulation FIX is continuously expressed and secreted into the bloodstream using the host cell gene transcription system, thereby promoting coagulation.

In December 2024, Belief released the results of a phase 3 clinical study at the 66th Annual Meeting of the American Society of Hematology, demonstrating that after 52 weeks of follow-up, the mean annualized bleeding rate (ABR) of the participants was 0.6, the average FIX activity reached 55.08 IU/dL and the average number of infusions of FIX drug decreased from 58.2 times/year before gene therapy treatment to 2.9 times/year after that. Twenty-one of the 26 participants had no bleeding events after treatment, and all participants had no serious adverse events.

The therapy was granted rare pediatric disease designation from the U.S. FDA and advanced therapy medical products designation from EMA last year.