SINO BIOPHARM: "Recombinant Human Coagulation Factor VIIA for Injection N01" approved for listing

According to the announcement from SINO BIOPHARM (01177), the group's developed "Recombinant Human Coagulation Factor VIIa for Injection N01" (trade name: Anqixin®) has received marketing approval from the National Medical Products Administration of China. It is indicated for the treatment of bleeding in adult and adolescent (aged 12 and above) patients with congenital hemophilia who have inhibitors to factor VIII or IX > 5 Bethesda units (BU). Anqixin is the first domestically approved recombinant human coagulation factor VIIa biological product in China.

The number of hemophilia patients worldwide is on a continuous upward trend, with statistics showing approximately 836,000 hemophilia patients globally, of which about 284,000 have severe hemophilia. The incidence of inhibitors in patients with severe hemophilia A is about 30%, while it is 3-13% in non-severe cases, and 1-6% in hemophilia B patients. Positive antibodies can lead to poor efficacy of traditional replacement therapies, severely affecting patients' quality of life. Recombinant human coagulation factor VIIa has become an important treatment option for patients with positive inhibitors due to its effective hemostatic action and unique bypass effect, and it has been widely used globally.

Anqixin is currently the only recombinant human coagulation factor VIIa in China that has confirmed efficacy and safety through Phase III clinical trials. Its approval is based on a multicenter, single-arm, open-label Phase III clinical study evaluating efficacy and safety in hemophilia patients with inhibitors. A total of 60 subjects were enrolled in the study, all of whom had used the investigational drug at least once, with 53 subjects experiencing a total of 559 bleeding events. An analysis of 551 assessable bleeding events showed a hemostatic efficacy rate of 88.93% (95% CI 86.01, 91.43).

In terms of production processes, recombinant human coagulation factor VIIa faces challenges such as complex structure, multiple degradation and oxidation sites, and various impurities. The group has innovatively developed proprietary processes for cell culture, separation and purification, and formulation, and has obtained two original patents: "Purification Method for Human Coagulation Factor VIIa (ZL202010330060.5)" and "Drug Composition of Recombinant Human Coagulation Factor VIIa (ZL202410278314.1)." The group has successfully conducted multiple batches of commercial production scaling, with good consistency in product batch quality, fully demonstrating the robustness of the production process.

As the first domestically approved injectable recombinant human coagulation factor VIIa product, Anqixin will provide patients with a more economical and high-quality treatment option. Previously, the group's recombinant human coagulation factor VIII for injection (trade name: Anhengji®) has also been approved for marketing. The group plans to cover the full course of treatment needs from routine replacement therapy to inhibitor management through a combination therapy scheme of Anhengji and Anqixin, which is expected to benefit a broader population of hemophilia patients