FDA Expands WILATE Prophylaxis Approval to Children Under 6 With von Willebrand Disease

The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application (sBLA) for WILATE (von Willebrand Factor/Coagulation Factor VIII Complex [Human]), expanding its indication for routine prophylaxis to reduce the frequency of bleeding episodes in children younger than six years with von Willebrand disease (VWD). The approval marks an important expansion of treatment options for pediatric patients at high risk of recurrent bleeding and fulfills a postmarketing requirement established under the Pediatric Research Equity Act (PREA).

The approval follows the FDA’s review of final clinical data from the WIL-33 study (NCT04953884), along with the required pediatric assessment submitted by Octapharma. The agency approved the supplemental application on July 2, 2026, extending the existing prophylactic indication to include patients below six years of age.

Addressing an unmet need in pediatric VWD

Von Willebrand disease is the most common inherited bleeding disorder, caused by deficiency or dysfunction of von Willebrand factor, a protein essential for platelet adhesion and stabilization of coagulation factor VIII. Children with severe forms of the disease may experience recurrent spontaneous bleeding that can affect quality of life and require long-term preventive therapy.

WILATE is a plasma-derived concentrate containing both von Willebrand factor and factor VIII. The replacement therapy restores deficient clotting proteins, helping prevent bleeding episodes in patients who require routine prophylaxis.

Pediatric study supported label expansion

The label expansion is based on final results from the Phase 3 WIL-33 pediatric study, which evaluated the safety and effectiveness of routine prophylaxis in children aged 2 years to younger than 6 years with von Willebrand disease. The FDA reviewed the complete study data and pediatric assessment before approving the expanded indication.

The supplemental application also fulfilled Postmarketing Requirement (PMR) #1, which was established following the December 2023 approval of WILATE for routine prophylaxis in older pediatric patients and adults. According to the FDA, the submission satisfies all outstanding postmarketing requirements and postmarketing commitments associated with this application. As a result, Octapharma is no longer required to submit annual postmarketing status reports for these commitments unless new obligations are issued in the future.

Regulatory milestone strengthens pediatric evidence

In its approval letter, the FDA confirmed that the pediatric study requirement under PREA has now been fulfilled for all age groups covered by the application. The agency also approved updated prescribing information and patient instructions reflecting the expanded indication. Final labeling must be submitted in Structured Product Labeling (SPL) format within 14 days of the approval date.

Clinical Implications

The expanded indication allows healthcare providers to prescribe WILATE for routine prophylaxis in children younger than six years with von Willebrand disease, broadening access to preventive therapy for one of the youngest patient populations affected by the disorder. The approval also completes Octapharma’s pediatric development commitments for this indication, providing additional clinical evidence supporting the use of WILATE across the full pediatric age spectrum.

What This Means for Patients

The FDA approval expands access to WILATE for children younger than six years with von Willebrand disease who require routine prophylaxis to prevent recurrent bleeding episodes. Until now, younger children had more limited evidence supporting preventive treatment. With this label expansion, healthcare providers can prescribe WILATE based on pediatric clinical data, offering families an FDA-approved option to reduce bleeding frequency, improve disease management early in life, and potentially lower the risk of bleeding-related complications.

Reference

July 2, 2026 Approval Letter – WILATE