Takeda's Recombinant von Willebrand Factor Vonicog alfa Approved in China as First-in-Class Therapy for Adult von Willebrand Disease Patients
Takeda
Biopharmaceutical Manufacturer
On August 5, the NMPA website announced that Takeda Pharmaceutical Company Limited submitted Voniglebin Alfa for Injection ( Vonicog alfa) Marketing Application (JXSS2300001/2) has been approved for use inAdult patients with von Willebrand disease (18 years and above)On-demand treatment and bleeding event control, as well as perioperative bleeding management. According to the Insight database,This is the first recombinant von Willebrand factor product approved in China.。
Screenshot source:NMPA Official Website
Von Willebrand disease is one of the common hereditary bleeding disorders, primarily due to von Willebrand factor (VWF) Gene mutations cause a reduction in the quantity or quality abnormalities of von Willebrand factor in plasma. As one of the important components of human plasma, von Willebrand factor mediates platelet adhesion to sites of vascular injury and serves as a carrier for coagulation factor VIII (FⅧ) carrier, which has the function of stabilizing coagulation factor VIII.
When von Willebrand factor is dysfunctional or deficient, blood cannot clot effectively, and patients may experience varying degrees of bleeding manifestations, significantly impacting their quality of life. Moreover, patients with von Willebrand disease who require surgery face a higher risk of bleeding as well as complications such as thrombosis, urgently necessitating more efficient bleeding management solutions to meet the critical clinical treatment needs of patients.
Vonicog alfa is an innovative recombinant von Willebrand factor (vWF) drug.rVWF）, it contains complete VWF multimers, including ultra-large multimers (ULMs), has a long half-life, can effectively achieve replacement therapy for vascular hemophilia, and provide patients with personalized methods for bleeding control.
In the Phase III on-demand treatment clinical trial, researchers evaluated Vonicog alfa for injection in adult patients with von Willebrand disease (18 Years of Age and Above) The hemostatic efficacy was studied in different dosing strategies, with or without recombinant coagulation factor FVIII, for on-demand treatment and bleeding event control. The study results showed,All bleeding events in subjects treated with Vonicog alfa, with or without recombinant coagulation factor FVIII, were controlled., the efficacy score is excellent (96.9%) or good (3.1%), and all bleeding events of all severity levels were controlled.
In addition, in another prospective, open-label, multi-center trial, researchers evaluated adult patients with severe vascular hemophilia (18 Years Old and Above) The hemostatic efficacy and safety of Vonicog alfa for injection, with or without recombinant coagulation factor FVIII, in elective surgical procedures. Study data shows, the overall hemostatic efficacy for subjects in major and minor surgeries was 100% (15/15）. Among them,60% of the surgeries showed excellent overall hemostatic efficacy, and 40% showed good overall hemostatic efficacy.。
Hemophilia is an important research area for Takeda. According to the Insight database, Takeda has received approval for 8 hemophilia drugs so far. In China, Takeda also has 4 hemophilia drugs on the market, including Vonicog alfa, which was approved today, as well as Susoctocog alfa (Acquired Hemophilia A), Recombinant Factor VIII - Advate (Hemophilia A), Nonacog Alfa (Hemophilia B, Bleeding, Coagulation Factor Deficiency, Hemophilia A）。
