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 2

"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]