On March 19, Belief Pharmaceuticals announced that its independently developed gene therapy drug, Xinjiuning™ (BBM-H901 injection, generic name: Popedacoji injection), has been officially approved by the Macau Drug Regulatory Authority for the treatment of adult patients with moderate to severe hemophilia B (congenital factor IX deficiency) . This product is the first hemophilia B gene therapy drug to be marketed in mainland China and Macau .
This drug utilizes recombinant adeno-associated virus (rAAV) to precisely deliver the highly active FIX gene into the patient's liver cells, resulting in sustained high expression of the FIX gene within the liver and its secretion into the bloodstream, where it exerts its procoagulant activity. Its design incorporates an engineered liver-targeting AAV843 capsid, a highly active FIX-Padua gene, a liver-specific strong promoter, and a double-stranded AAV design, aiming to achieve rapid, efficient, and sustained gene expression after intravenous administration.
The efficacy and safety of this drug are supported by multiple studies:
In a multicenter, single-arm phase III clinical trial (n=26) , 52-week follow-up results showed:
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The annualized bleeding rate (ABR) was 0.60 (95% CI: 0.18-1.99) , which was significantly lower than the superiority margin (mean ABR of 5.0 for hemophilia B patients in China receiving prophylactic treatment) .
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At 52 weeks, the average FIX:C ratio was 55.1 (SD35.9) IU/dL (OSA, SynthAsil) .
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The annualized average number of FIX infusions decreased from 58.20 infusions/year before treatment (SD 30.67) to 2.90 infusions/year after treatment (SD 10.71) ;
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The number of joint bleeding episodes decreased significantly, and the number of target joints dropped to 0.9 .
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In IIT, the results of a 5-year follow-up showed:
By the last visit [median 210 weeks (range 159–270 weeks) ], 70% of subjects had FIX:C > 35 IU/dL, of which 40% had > 50 IU/dL (using the Actin FSL APTT reagent in a single-phase method) .
Regarding safety, no adverse reactions of grade ≥3 occurred in any of the subjects, no serious adverse events (SAEs) were reported, and no inhibitors were produced. All patients in the above studies are still under ongoing follow-up.
Meanwhile, another rAAV gene therapy drug from Believe Pharmaceuticals targeting hemophilia A recently completed the dosing of the first subject in its Phase III registration clinical trial. This candidate drug delivers the gene expressing optimized coagulation factor VIII (FVIII) to the patient's liver cells via intravenous infusion, aiming to achieve long-term efficacy with a single dose.

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