Belief Biomed and Takeda Announce NMPA Acceptance of NDA for Gene Therapy BBM-H901 in Adults with Hemophilia B

Jul 24, 2024 17:29 CST Updated 17:29

Takeda

Biopharmaceutical Manufacturer

▎Edited by the WuXi AppTec content team

Today (July 24),Belief BioMed and Takeda(Takeda) Co.,Announced that the China National Medical Products Administration (NMPA) has formally accepted the application for BBM-H901 Injection for the treatment ofAdult patients with Hemophilia BThe new drug application (NDA). According to a press release from Belief BioMed, BBM-H901 is the first product independently developed and manufactured by the company, and also the first gene therapy drug for genetic diseases to submit a new drug application in China. The product will be commercially promoted in mainland China, Hong Kong, and Macao by Takeda Pharmaceutical Company Limited.

There are still unmet medical needs in the Chinese hemophilia B patient population.BBM-H901 Injection is developed and manufactured by Belief BioMed.Recombinant Adeno-Associated Virus (AAV) Gene Therapy Drugs,Administered intravenouslyHuman Coagulation Factor IX (FIX)Gene therapy introduced into Hemophilia B patients for continuous expression, enhancing and maintaining the coagulation factor level over the long term, used to prevent bleeding in adult Hemophilia B patients.。

In 2022, BBM-H901 injection was included in the breakthrough therapy category by the NMPA. The results of its investigator-initiated clinical study (IIT) were successively published in the prestigious international journals *The Lancet Haematology* and *The New England Journal of Medicine*, and were presented at the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress.Oral ReportFormat display. Research shows,During the long-term follow-up of 3.0-4.5 years,FIX was stably and highly expressed in 9 subjects, with no bleeding events occurred, an annualized bleeding rate (ABR) of 0, and complete cessation of exogenous FIX product replacement therapy.。

According to the news release from Belief BioMed, thisThe submission of BBM-H901 for marketing authorization is based onA multi-center, single-arm, open-label, single-dose registration clinical study aimed at evaluatingSingle Intravenous InfusionBBM-H901 Injection inPatients with Hemophilia B aged ≥18 years and endogenous coagulation factor IX (FⅨ) activity ≤2 IU/dL (i.e., ≤2%)The safety and efficacy in China. To date, all subjects in the Phase 3 confirmatory study have completed the 52-week visit after dosing, and the safety and efficacy of this registration study have met expectations.

It is reported that, in 2023, Takeda China announced an exclusive commercialization cooperation agreement with Belief BioMed in the field of Hemophilia B. Both parties will combine their respective strengths and resources to accelerate the commercialization process of BBM-H901 injection, helping Hemophilia B patients gain access to innovative treatment options and jointly promoting the industrial development of gene therapy.

References:

[1]First in China! Belief BioMed's New Drug Application for BBM-H901 Injection, a Gene Therapy for Hemophilia B, Accepted by the National Medical Products Administration!. Retrieved Jul 24, 2024, from https://mp.weixin.qq.com/s/lclVflAM-h2A5apndZO7FQ

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