Takeda's New Hemophilia Drug Vonicog Alfa Nears Priority Review in China for von Willebrand Disease

Intelligent Finance APP learned on January 18 that the latest announcement on the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration showed that the listing application of Vonicog Alfa for injection, submitted by Takeda (TAK.US), is proposed to be included in the priority review. The indication is: for adult patients diagnosed with von Willebrand Disease (VWD), including on-demand treatment and control of bleeding events, as well as perioperative bleeding management. Public data shows that Vonicog Alfa for injection is a recombinant von Willebrand factor, which has been approved for marketing in many overseas regions such as the United States and Japan.

Screenshot source: CDE official website

It is reported that von Willebrand Factor (VWF), as one of the important plasma components in the human body, mediates platelet adhesion to vascular injury sites and acts as a carrier for coagulation factor VIII (FVIII), stabilizing coagulation factor VIII. Vonicog alfa for injection is a recombinant von Willebrand factor. It contains full-length VWF multimers, including ultra-large multimers, has a long half-life, and can effectively achieve replacement therapy for von Willebrand disease while providing patients with personalized bleeding control methods.

According to a press release previously issued by Takeda, vonicog alfa for injection has been approved for marketing in the United States, Canada, the United Kingdom, Switzerland, Australia, and Japan. It is used for on-demand treatment and bleeding event control in adult patients with von Willebrand disease, as well as perioperative bleeding management.

Notably, in the Phase 3 on-demand treatment clinical trial, researchers evaluated the hemostatic efficacy of injectable vonicog alfa in adult patients with von Willebrand disease and investigated different dosing strategies, with or without recombinant factor VIII, for on-demand treatment and bleeding event control. Trial data showed that all subjects treated with injectable vonicog alfa, with or without recombinant factor VIII, had their bleeding events controlled, with efficacy ratings of excellent or good, and all bleeding events of varying severity were successfully managed.

In another prospective, open-label, multicenter trial, researchers also evaluated the hemostatic efficacy and safety of vonicog alfa for injection, with or without recombinant factor VIII, in adult patients with severe von Willebrand disease undergoing elective surgical procedures. The trial results showed that the overall hemostatic efficacy was 100% (15/15) for both major and minor surgeries. Specifically, 60% of the surgeries demonstrated excellent overall hemostasis, while 40% showed good overall hemostasis.