Takeda's Recombinant von Willebrand Factor (rVWF) Vonicog alfa NDA Accepted for Review in China

ShanghaiJanuary 7, 2023PR Newswire -- Takeda China announced today that its innovative drug, recombinant von Willebrand Factor (rVWF) for injection, Vonicog alfa^[1]The New Drug Application (NDA) has been officially accepted by the Center for Drug Evaluation of the National Medical Products Administration, intended for on-demand treatment and bleeding event control in adult patients with von Willebrand Disease (18 years and above), as well as perioperative bleeding management. Vonicog alfa^[1]Is the world's first and only recombinant von Willebrand factor product to date. ^[2], is expected to bring a new treatment option for adult patients with vascular hemophilia in China.

Von Willebrand disease is one of the common hereditary bleeding disorders, primarily caused by mutations in the von Willebrand factor (VWF) gene, leading to a reduced quantity or abnormal quality of von Willebrand factor in plasma.^[3]As one of the important plasma components in the human body, von Willebrand Factor (VWF) mediates platelet adhesion to vascular injury sites and acts as a carrier for coagulation factor VIII (FVIII), stabilizing coagulation factor VIII.^[3]。

When von Willebrand factor is dysfunctional or deficient, blood cannot clot effectively, and patients may experience varying degrees of bleeding manifestations, primarily skin and mucosal bleeding, characterized by petechiae, ecchymoses, epistaxis, gingival bleeding, menorrhagia in women, and severe cases may involve internal bleeding.^[4], which has a serious impact on the quality of life. In addition, patients with vascular hemophilia who require surgery face higher risks of bleeding and complications such as thrombosis, urgently needing more efficient bleeding management solutions to meet the critical clinical treatment needs of patients. ^[5]。

Vonicog alfa for Injection^[1]Contains complete VWF multimers, including ultra-large multimers (ULMs), with a long half-life, effectively achieving replacement therapy for von Willebrand disease and providing patients with personalized bleeding control methods.^[6]. In the Phase 3 on-demand treatment clinical trial, injectable Vonicog alfa was evaluated.^[1]The hemostatic efficacy for adult patients (18 years and above) with von Willebrand disease was studied, evaluating different on-demand treatment strategies with or without recombinant factor VIII for the control of bleeding episodes. The results showed that all injectable Vonicog alfa...^[1]Bleeding events were controlled in subjects with or without concomitant use of recombinant coagulation factor FVIII, with efficacy rated as excellent (96.9%) or good (3.1%), and all bleeding events of any severity were successfully managed.^[6]。

In addition, in another prospective, open-label, multicenter trial, the use of Vonicog alfa for injection was evaluated in adult patients (18 years and older) with severe von Willebrand disease undergoing elective surgical procedures.^[1]Hemostatic efficacy and safety with or without recombinant coagulation factor FVIII: Study results showed that the overall hemostatic efficacy for major and minor surgeries was 100% (15/15). Among these, the overall hemostatic efficacy was rated as excellent for 60% of the surgeries and good for 40% of the surgeries.^[6]。

Currently, Vonicog alfa for injection^[1]Has received marketing authorization in the United States, Canada, the United Kingdom, Switzerland, Australia, and Japan for on-demand treatment and bleeding episode control in adult patients with von Willebrand Disease (18 years and older), as well as perioperative bleeding management.^[7],[8],[9]. As an innovative drug in the field of von Willebrand disease, the marketing authorization application for Vonicog alfa for injection has been officially accepted in China.^[1]Chinese Patients with Von Willebrand Disease to Benefit Soon.

With over 70 years of rich experience and heritage in the field of rare bleeding and coagulation disorders, Takeda Pharmaceutical Company Limited boasts a wide-ranging, diversified, and highly differentiated portfolio, offering transformative and personalized treatment options for patients with hemophilia and other blood disorders. Guided by the philosophy of "patient-first," Takeda plans to introduce several innovative therapies in China within the next five years, covering areas such as von Willebrand Disease, acquired hemophilia, and thrombotic thrombocytopenic purpura, further addressing unmet needs of patients with rare bleeding and coagulation disorders in China.

Statement

1. This article aims to convey cutting-edge pharmaceutical information and does not constitute a recommendation or promotion of any drug or treatment plan.
2. If you want to learn more about disease knowledge or drug and treatment-related information, please consult a healthcare professional.
3. The Vonicog alfa for injection mentioned in this article has not yet been launched in China.

^{[1] This name is the English generic name submitted for approval and is subject to the final approval result.
[2] https://www.pharmatimes.com/news/us_expands_use_of_shires_vonvendi_1232279(As of January 2023)
[3] Thrombosis and Hemostasis Group, Hematology Branch of Chinese Medical Association. Chinese Expert Consensus on the Diagnosis and Treatment of Von Willebrand Disease (2012 Edition)［J］. Chinese Journal of Hematology, 2012, 33(11): 980-981. DOI: 10.3760/cma.j.issn.0253-2727.2012.11.023.
[4] Hemostasis and Thrombosis Group of the Hematology Society of Chinese Medical Association. Chinese Guidelines for the Diagnosis and Treatment of Von Willebrand Disease (2022 Edition)[J]. Chinese Journal of Hematology, 2022, 43(1):6.
[5] Gill JC, Castaman G, Windyga J et al. "Hemostatic efficacy, safety and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease." Blood. 2015;126(17):2038-2046.
[6] VONVENDI [von Willebrand factor (recombinant)] Prescribing Information. 
[7] https://www.takeda.com/newsroom/shire-news-releases/2016/nbtjyp/
[8] https://www.takeda.com/newsroom/shire-news-releases/2018/s7os8o/
[9] https://www.ema.europa.eu/en/medicines/human/paediatric-investigation-plans/emea-001164-pip01-11-m04}