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

LONDON, Sept. 25, 2020 /PRNewswire/ -- Takeda UK Ltd. is pleased to announce that following a review meeting in July 2020, the NHS England Clinical Priorities Advisory Group (CPAG) has provided a positive recommendation for the routine commissioning of VEYVONDI^®?(vonicog alfa), the first and only recombinant von Willebrand factor (VWF) treatment for adults with von Willebrand disease (VWD).^[2]

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 commissioned for the treatment of haemorrhage and surgical bleeding, and prevention of surgical bleeding, in adults (aged 18 years or older) with a confirmed diagnosis of VWD, in the following circumstances:^[3]

• when desmopressin with or without tranexamic acid treatment is ineffective or not indicated (based on UK clinical practice); AND
• when VWF activity levels are <50 IU/dl OR diagnosis is type 2N VWD; AND
• there is no evidence of inhibitors to VWF

This CPAG decision means that adult patients with VWD and the Haematologists who treat them will now have access to vonicog alfa (within its licenced indication) as an alternative to plasma-derived von Willebrand factor (pdVWF) concentrates.^[3] The current standard of care for treating VWD-related bleeding episodes and treating and preventing bleeding during surgery, is for pdVWF concentrates to be given if response to desmopressin is inadequate or not indicated.^[3]

Jo Traunter, Haemophilia Society UK Trustee, said: "The Haemophilia Society welcomes the announcement by NHS England that the first recombinant treatment for adults living with VWD will be made available. Until now, adults living with VWD have not had a recombinant treatment option, so we are delighted to share this news with our members. It is a significant development in access to treatment and shows that our successful 'Recombinant for All campaign' which started almost 20 years ago is just as important for our community today. Research suggests that as many as 9 out of 10 people with VWD have not been diagnosed.^[1] Their bleeding symptoms may be mild and it's not until an event such as an injury, or during surgery, when the blood has difficulty forming a clot that the condition is picked up and diagnosed. It is vital that we continue to raise awareness of this condition and its symptoms and ensure the voice of our community is heard."

"The availability of vonicog alfa provides clinicians, for the first time, with a recombinant treatment option for managing adult patients with VWD in England," said Dr Carolyn Millar, Consultant Haematologist specialising in Haemostasis and Thrombosis, Imperial College London. "As it contains only trace amounts of recombinant FVIII, and since many situations requiring VWF replacement do not also require exogenous factor VIII, this profile offers clinicians the flexibility to dose vonicog alfa with or without rFVIII based on the patient's individual needs with appropriate monitoring of levels. Furthermore, the half-life of vonicog alfa ranges between 18 and 23 hours and, due to its manufacturing process, it also contains ultra-large multimers in addition to all of the multimers found in circulating plasma."^[2]

"We're delighted by this announcement from NHS England. As a leader in rare disease, Takeda is committed to advancing standards of care and outcomes for people living with rare bleeding disorders. Clinicians now have the option to routinely prescribe a recombinant treatment for VWD and this decision represents a significant development for adult patients with VWD," said Jon Neal, Managing Director, Takeda UK and Ireland.

About von Willebrand Disease

VWD is the most common inherited bleeding disorder, affecting up to one percent of the world's population, however research shows that as many as nine out of 10 people with VWD have not been diagnosed.^[1]

VWD is caused by a deficiency or dysfunction of VWF, a blood protein required for proper clotting.^[4] Occurring equally in men and women, people with VWD have difficulty forming a blood clot and often have bleeding from the mucous membranes.^[4],[5] Bleeding caused in people living with VWD is unpredictable and varies greatly.^[5] Many people living with VWD may not know that they have the condition.^[6] This may be because symptoms can begin at any age, change over time and can range from being very mild and barely noticeable to frequent and severe.^[6] Symptoms can include nosebleeds, bleeding from the gums, easy bruising, and heavy menstrual bleeding. In addition, people with the condition may bleed easily after injury, childbirth, and surgery.^[4],[6] According to the degree of deficiency or defect of VWF observed, VWD is classified into three main phenotypes, Type 1, Type 2 and Type 3.^[5]

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]