The FDA Approves a Treatment for Patients with Von Willebrand Disease Undergoing Surgery

The US Food and Drug Administration (FDA) has just approved the drug Vonvendi for use in adult patients with von Willebrand disease who are undergoing surgery. This is an extension of previous FDA approval of Vonvendi for treatment of bleeding episodes in patients with the same condition. The full press release can be found here, at Globe Newswire.
Von Willebrand disease (VWD) is the most common bleeding disorder in the United States, where it is thought to affect approximately 1 in every 100 people. It is a genetic disease that leads to patients lacking a functioning version of a protein involved in blood clotting, called a von Willebrand factor (VWF). In people without the disease the VWF protein blinds to other types of proteins in the blood, including platelets and factor VIII, to form blood clots. This process doesn’t effectively occur in patients with VWD, and as a result they are likely to experience symptoms such as frequent and heavy nosebleeds, heavy menstruation, frequent bruising, and excessive bleeding from surgery or childbirth.
There are currently several treatment options for patients with VWD, including vasopressin to increase VWF levels, and other drugs that stop blood clots from breaking down. The drug Vonvendi, which has just had its approve usages extended, is currently the only available recombinant VWF treatment available. This means that the manufacturing process involves separating out specific proteins from a blood sample, and then concentrating them to produce the treatment. It is also the only product to contain VWF only, rather than VWF in combination with factor VIII. This allows for individualised patient treatment, since not all patients with the condition lack the factor VIII protein. Vonvendi can be left without factor VIII, or combined with it, depending on the individual patient needs. This is a significant advantage of the treatment since excessive levels of factor VIII have been linked to complications such as excessive blood clots.

The FDA’s approval of Vonvendi for use with surgery in patients with VWD follows a successful Phase 3 clinical trial of fifteen participants with severe VWD who underwent surgery and were treated with Vonvendi. The patient outcomes showed that the drug was effective; patient bleeding was found to have reduced.

The use of Vonvendi for surgery will give doctors and patients another option for treating VWD, and allow them to do it in a more personalized way. Since patients with the condition are at a higher than average risk of severe bleeding from operations, effective and appropriate treatment options are important.

Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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