Takeda Pharmaceutical Company Limited (TYO: 4502 / NYSE: TAK), a leading Japanese pharmaceutical company, has announced that it has received marketing approval from China’s National Medical Products Administration (NMPA) for vonicog alfa, a recombinant von Willebrand factor (rVWF), for the on-demand treatment and control of bleeding events in adult patients with Von Willebrand disease (VWD) aged 18 and above, as well as for perioperative bleeding management. Known globally as Veyvondi, vonicog alfa is part of the rare disease portfolio that Takeda acquired from Shire in 2019.
VWD is a hereditary bleeding disorder characterized by reduced or abnormal levels of von Willebrand factor (VWF) in the plasma due to genetic mutations, leading to frequent bleeding events from a young age, particularly on the skin and mucous membranes. Vonicog alfa is currently the world’s first and only rVWF produced using genetic recombination technology, offering a complete polymer structure and an extended half-life. This allows for precise alternative treatment for hemophilia and personalized bleeding control methods for patients, without the risk of blood-borne infectious diseases.
A Phase III on-demand treatment study evaluated the hemostatic efficacy of vonicog alfa in adult patients (aged 18 and older) with severe hemophilia. The study explored different administration strategies for on-demand treatment and bleeding event control with or without recombinant coagulation factor FVIII. Results indicated that all subjects who received vonicog alfa, with or without recombinant coagulation factor FVIII, achieved 100% effective control of bleeding events, with 96.9% rated as excellent and 3.1% as good. Additionally, a prospective, open-label, multicenter trial assessed the hemostatic efficacy and safety of vonicog alfa in combination with or without recombinant coagulation factor FVIII during elective surgical procedures in adult patients with severe hemophilia (aged 18 and older). The results showed that the overall hemostatic efficacy in major and minor surgeries was 100% (15/15), with 73.3% of surgeries having excellent overall hemostatic efficacy and 26.7% good. In both studies, vonicog alfa demonstrated a favorable safety profile.- Flcube.com
