Vonvendi is now the only recombinant von Willebrand factor replacement therapy with indications in adults and children with von Willebrand disease, according to the company.

Previously, the product was approved for on-demand and perioperative use in adults and for routine prophylaxis only in adults with severe type 3 disease receiving on-demand therapy.

von Willebrand disease, the most common inherited bleeding disorder, affects more than 3 million people in the US and results from low levels or dysfunctional von Willebrand factor (VWF), impairing normal clot formation. In clinical trials, most non-surgical bleeds were treated with one infusion in adults (157 of 192) and children (80 of 104). The reported half-life was 22.6 hours in adults and 14.3 hours in children.

Key safety considerations include thromboembolic events (eg, disseminated intravascular coagulation, venous thrombosis, pulmonary embolism, myocardial infarction, stroke); hypersensitivity and infusion-related reactions; and potential development of neutralizing antibodies to VWF and factor VIII. Monitoring of VWF ristocetin cofactor and factor VIII activity is recommended to avoid sustained excessive activity levels, particularly when recombinant factor VIII is coadministered. Vonvendi is contraindicated in patients with life-threatening hypersensitivity to product components, including trace mouse immunoglobulin G and hamster proteins. The most common adverse reactions observed in greater than or equal to 2% of patients were headache, vomiting, nausea, dizziness, and generalized pruritus.

The approval was supported by three clinical trials in adults and children and by supportive real-world data. The company reported related pediatric regulatory submissions in Japan and the European Union.

Clinicians should consult the full Prescribing Information for dosing, monitoring, and perioperative guidance.

Source: Takeda Pharmaceutical Company