The FDA has expanded approval of Wilate for routine prophylaxis in children under 6 with von Willebrand disease (VWD), making it the first von Willebrand factor concentrate approved for all ages and all disease types. The decision was backed by phase 3 trial data showing a low annual bleeding rate and strong tolerability in toddlers and young children. Pediatric hematologists now have an FDA-approved prophylactic option for this vulnerable age group.
The FDA has expanded its approval of Wilate (von Willebrand factor/human coagulation factor VIII complex, Octapharma) to include routine prophylaxis for children younger than 6 years with von Willebrand disease (VWD). Previously approved for adults and children aged 6 and older, Wilate is now the first VWF concentrate with an indication spanning all patient ages and all VWD types — a meaningful milestone for pediatric hematology.
The approval was based on data from the phase 3 WIL-33 trial, which enrolled 12 children under age 6 (median age: 2 years) with severe VWD. Patients received Wilate two to three times per week for 12 months, and the treatment proved both safe and effective.
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Why it matters: Until now, there was no FDA-approved prophylactic option for children under 6 with VWD — a gap that left pediatric hematologists without evidence-based tools for this high-risk group. This approval aligns treatment options for young children with those available to older patients, potentially reducing the burden of severe bleeding episodes early in life.
