PARAMUS, N.J. | July 8, 2026
Octapharma USA has announced that the U.S. Food and Drug Administration (FDA) has expanded the approval of wilate® (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) for routine prophylaxis to reduce the frequency of bleeding episodes in children younger than six years of age with von Willebrand Disease (VWD). The regulatory milestone makes wilate® the first von Willebrand Factor (VWF) concentrate approved for routine prophylactic treatment across all age groups and all forms of VWD, significantly expanding treatment options for pediatric patients with the most common inherited bleeding disorder. The expanded indication is supported by positive data from the Phase III WIL-33 clinical trial, which demonstrated that prophylactic treatment with wilate® was safe, effective, and well tolerated in children under six years of age. The FDA decision represents an important advancement in pediatric hematology by providing clinicians with the first approved preventive therapy for young children at risk of recurrent bleeding episodes.
FDA Approval Expands Preventive Treatment for Young Children With VWD
The expanded FDA approval addresses a significant unmet medical need for pediatric patients with severe von Willebrand Disease, particularly children younger than six years of age who previously lacked an FDA-approved option for routine prophylaxis. Von Willebrand Disease (VWD) is the most common inherited bleeding disorder and is caused by a deficiency or dysfunction of von Willebrand Factor (VWF), a protein essential for normal blood clotting. Patients with severe disease frequently experience spontaneous bleeding episodes that may require repeated medical intervention and can significantly affect quality of life.
With the expanded label, wilate® is now approved for routine prophylaxis in both adult and pediatric patients of all ages, allowing physicians to reduce bleeding frequency through scheduled preventive treatment rather than relying solely on treatment after bleeding occurs. The approval also aligns pediatric treatment options with current evidence-based clinical guidelines recommending prophylaxis for patients experiencing severe and frequent bleeding episodes.
Phase III WIL-33 Trial Demonstrates Strong Safety and Efficacy
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The FDA decision is based on results from the international Phase III WIL-33 clinical trial, an open-label, multicenter study evaluating 12 pediatric patients younger than six years with severe VWD. During the study, patients received wilate® two to three times weekly at recommended doses over a 12-month treatment period. Clinical findings demonstrated a low annual bleeding rate, with the majority of bleeding events classified as minor and 95.6% of treated bleeding episodes successfully controlled with a single infusion. Investigators also reported that prophylaxis with wilate® was well tolerated, with no thrombotic events, no Factor VIII accumulation, and no unexpected safety concerns observed throughout the study.
The WIL-33 findings complement the previously completed WIL-31 study, the largest prospective investigation of wilate® prophylaxis in older children and adults, confirming consistent efficacy across all pediatric age groups. These clinical results provide important pharmacokinetic, efficacy, and safety evidence supporting long-term preventive treatment in very young patients with severe bleeding disorders.
Strengthening Pediatric Hematology Through Plasma-Derived Therapies
The expanded FDA approval further strengthens Octapharma’s leadership in plasma-derived therapies and reinforces the company’s commitment to advancing innovative treatments for patients living with rare hematologic disorders. Wilate® is indicated for on-demand treatment of bleeding episodes, perioperative bleeding management, and routine prophylaxis in patients with von Willebrand Disease, while also being approved for selected indications in Hemophilia A. As one of the world’s largest manufacturers of human plasma-derived therapies, Octapharma continues investing in clinical research focused on improving outcomes across hematology, immunotherapy, and critical care.
The latest regulatory milestone not only expands access to preventive treatment for young children with VWD but also provides physicians with an evidence-based therapeutic option capable of reducing bleeding complications during the earliest years of life. By combining strong clinical evidence with regulatory approval, wilate® is expected to play an increasingly important role in advancing pediatric hematology, rare disease management, and personalized bleeding disorder care, helping improve long-term outcomes for patients and families affected by von Willebrand Disease.
Source: Octapharma press release
