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."