The US FDA issued an approval to expand the label of Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) for routine prophylaxis in children younger than 6 years with von Willebrand disease (VWD). The decision closes a gap that has left pediatric hematologists without a formally approved option for this age group. Switzerland-based Octapharma’s US subsidiary now holds the only VWF concentrate label covering prophylactic treatment across all ages and all forms of VWD — the most common inherited bleeding disorder.
The label expansion, which covers routine prophylaxis to reduce bleeding episode frequency in pediatric patients under six, is administered intravenously two to three times per week at 30 to 50 international units (IU)/kg. Wilate delivers both von Willebrand Factor and coagulation Factor VIII derived from human plasma, simultaneously addressing the dual deficiency characteristic of VWD: insufficient VWF activity impairs platelet adhesion and primary hemostasis, while co-administered FVIII supports the coagulation cascade. The plasma-derived formulation carries standard warnings applicable to human plasma products, including risks of hypersensitivity, thromboembolic events, and infectious agent transmission.
The approval rests on the Phase III WIL-33 trial (NCT04953884), an open-label, prospective, international, multicenter study enrolling 12 patients under six years of age with severe VWD (VWF ristocetin cofactor activity <20%). The primary endpoint was the total annualized bleeding rate during prophylaxis over 12 months. The observed annualized bleeding rate was 4.6 ± 6.1; of 56 bleeding episodes recorded, 98.2% were classified as minor, and 95.6% of treated episodes required only a single infusion. No thrombotic events or FVIII accumulation were reported. The WIL-33 findings were consistent with those from the earlier WIL-31 study, the largest prospective prophylaxis study conducted in adults and children aged six and older with VWD.