FDA Expands Approval to wilate for Prophylaxis in All Types of VWDr

Octapharma USA’s wilate, von Willebrand factor (VWF)/coagulation factor VIII complex (human) lyophilized powder for solution for intravenous injection, has been given expanded approval by the U.S. Food and Drug Administration (FDA) for routine prophylaxis to reduce 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 VWF concentrate indicated for prophylactic treatment across all forms of VWD.

Approval was based on Octapharma’s WIL-31 study, a prospective, noncontrolled, international, multicenter Phase III 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 percent 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 percent reduction in the mean total ABR compared with on-demand treatment during the prior study. The median spontaneous ABR decreased by 95 percent. Importantly, no serious drug-related adverse events or thrombotic events were observed during the study.

“Long-term prophylaxis with VWF concentrate, as compared to on-demand treatment for bleeding, is recommended for patients with severe VWD,” said 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 lifesaving for many patients. Increased use of VWF prophylaxis in VWD patients may lead to improved patient care and a reduced burden of disease.”