The US Food and Drug Administration (FDA) has approved the use of Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) for routine prophylaxis to reduce the frequency of bleeding episodes in paediatric patients with von Willebrand Disease (VWD) who are younger than six years of age.
This approval expands access to the first von Willebrand Factor (VWF) concentrate indicated for prophylactic treatment across all ages and forms of VWD, the most common inherited bleeding disorder.
Octapharma USA announced that pediatric patients, including children younger than 6 years of age, with von Willebrand Disease (VWD) now have access to routine prophylaxis to help reduce the frequency of bleeding episodes. The FDA’s expanded approval for wilate® (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) Lyophilized Powder for Solution for Intravenous Injection, expands access to the first VWF concentrate indicated for routine prophylaxis in both adult and pediatric patients.
The expanded label is based on data from the phase 3 clinical trial, an international study to research the safety and efficacy of VWF prophylaxis in children with VWD under the age of six.
The open-label WIL-33 trial enrolled and treated 12 patients, all under the age of 6 and diagnosed with severe VWD. Wilate was administered two to three times per week at a recommended dose of 30 to 50 international units (IU)/kg over 12 months. The primary endpoint was the total annualised bleeding rate (TABR) during prophylaxis, with safety and tolerability assessed throughout the study.
