The U.S. Food and Drug Administration (FDA) has expanded the approval for Octapharma USA’s wilate, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection.
The approved label now includes routine prophylaxis aimed at reducing the frequency of bleeding episodes in adults and children aged 6 and older with any type of von Willebrand disease (VWD), the most prevalent bleeding disorder in the United States. Wilate is the first von Willebrand factor (VWF) concentrate indicated for prophylactic treatment across all forms of VWD, marking a significant milestone in the field.
“Long-term prophylaxis with VWF concentrate, as compared to on-demand treatment for bleeding, is recommended for patients with severe VWD,” according to Shveta Gupta, MD, a specialist in pediatric hematology and oncology with The Haley Center for Children’s Cancer and Blood Disorders at Orlando Health Arnold Palmer Hospital for Children. “The approval of wilate for VWD prophylaxis is a welcome new treatment option that can be life-saving for many patients. Increased use of VWF prophylaxis in VWD patients may lead to improved patient care and a reduced burden of disease.”
The FDA approval is supported by Octapharma’s WIL-31 study, a prospective, non-controlled, international, multicenter phase 3 trial that investigated the efficacy and safety of wilate prophylaxis over 12 months in people aged 6 and older with severe VWD of any type.
All WIL-31 patients received on-demand treatment with wilate during a previous six-month, prospective, observational study (WIL-29). Patients who experienced at least six bleeding episodes (BEs), excluding menstrual bleeds, with at least two of these BEs treated with a VWF-containing product, were eligible to enter WIL-31. Patients in WIL-31 received wilate prophylaxis two to three times per week at a dose of 20-40 IU/kg, for 12 months.
The clinical trial’s primary purpose was to investigate whether prophylaxis with wilate lowered the mean total annualized bleeding rate (ABR) by more than 50% compared to the six months of on-demand treatment. Secondary goals were to measure spontaneous ABR and treatment-emergent adverse events.
Researchers reported an 84% reduction in the mean total ABR compared with on-demand treatment during the prior study. The median spontaneous ABR decreased by 95%. Importantly, no serious drug-related adverse events or thrombotic events were observed during the study.
Von Willebrand disease affects up to 1% of the U.S. population, equating to about 3.3 million individuals. The disease, occurring equally among men and women, is often noticed more by women due to heavy or abnormal bleeding during menstrual periods and after childbirth. VWD includes three major types – Type 1, Type 2, and Type 3, the most severe form. The expanded wilate label provides a treatment option for a significant patient population.
“Wilate prophylaxis for adults and children aged 6 and older with VWD promises to be life-changing for many patients,” stated Octapharma USA President Flemming Nielsen. “Patients have been forced to live with far too many bleeding episodes while receiving on-demand treatment. Octapharma is pleased to offer patients a new therapy option that can greatly improve their quality of life.”