First and only recombinant von Willebrand factor treatment for adults with von Willebrand Disease, the most common inherited bleeding disorder[1], recommended for routine commissioning via NHS England - Seite 3

About VEYVONDI in the UK

Vonicog alfa (VEYVONDI) was granted European Marketing Authorisation in August 2018 and is indicated in adults (aged 18 and older) with VWD, when desmopressin treatment alone is ineffective or not indicated for the

• treatment of haemorrhage and surgical bleeding
• prevention of surgical bleeding

Vonicog alfa should not be used in the treatment of Haemophilia A.^[2]

Vonicog alfa is a recombinant human VWF that works in the body in the same way as natural VWF.^[2] It replaces the deficient or defective protein, helping the blood to clot and giving temporary control of bleeding.^[2] It is administered as an intravenous infusion for treating on-demand bleeding episodes and for preventing and treating bleeding during surgery when desmopressin treatment is ineffective or not indicated.^[2] Vonicog alfa would be an alternative treatment option to pdVWF concentrates for an adult patient with VWD.^[3]

Following this NHS England CPAG routine commissioning reimbursement decision, vonicog alfa will be reimbursed in England from September 2020.^[3]

About Takeda UK Ltd.

Takeda Pharmaceutical Company Limited is headquartered in Japan and is a global, values-based, R&D-driven biopharmaceutical leader committed to translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Neuroscience and Rare Diseases. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

Additional information about Takeda UK Ltd. is available through its corporate website, www.takeda.com/en-gb.

References:

^1. World Federation of Hemophilia [Internet]. Von Willebrand Initiative Program. Available at: https://www.wfh.org/en/our-work-global/vwd-initiative-program. [Last accessed September 2020]
^2. Veyvondi Summary of Product Characteristics. Electronic Medicines Compendium [Internet]. Available at: https://www.medicines.org.uk/emc/product/11233/smpc [Last accessed September 2020]
^3. Clinical Commissioning Policy: Vonicog alfa for the treatment and prevention of bleeding in adults with von Willebrand disease [Internet] Available at: https://www.england.nhs.uk/wp-content/uploads/2020/09/1709-vonicog-alfa-clinical-commissioning-policy.pdf [Last accessed September 2020]
^4. Nichols WL, et al (2008) 'von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA)' Haemophilia 14:171-232
^5. Leebeek FW, Eikenboom JC (2016) 'Von Willebrand's Disease' The New England Journal of Medicine Nov;375:2067-80
^6. World Federation of Haemophilia [Internet]. What is von Willebrand Disease? Available at: http://www1.wfh.org/publication/files/pdf-1330.pdf [Last accessed September 2020]