FDA Expands Approval for VWD Prophylaxis in Children Younger Than 6

The FDA has approved von Willebrand Factor/Coagulation Factor VIII Complex (Human) (wilate; Octapharma USA) for routine prophylaxis to reduce bleeding episode frequency in children younger than 6 years with von Willebrand disease (VWD). The agency's decision extends the drug's reach across the full age spectrum of the most common inherited bleeding disorder.¹

Wilate is now the only von Willebrand factor (VWF) concentrate cleared for prophylactic use in both adults and children with all forms of VWD. The expanded label follows the phase 3 WIL-33 trial (NCT04953884), an open-label, international study designed to evaluate the safety and efficacy of VWF prophylaxis in children under age 6.

"Evidence-based treatment guidelines recommend prophylactic treatment for VWD patients with a history of severe, frequent bleeds," Flemming Nielsen, president of Octapharma USA, said in a statement. "Until now, pediatric hematologists have not had an FDA-approved option for routine prophylaxis in young children with VWD. With this expanded indication, children younger than 6 years of age can now receive routine prophylaxis to reduce the frequency of bleeding episodes, consistent with treatment options available for older children and adults."

What the WIL-33 Trial Showed

WIL-33 enrolled 12 children, all younger than 6 years, with severe VWD, defined as VWF ristocetin cofactor activity under 20%. At baseline, the median age was 2 years, half the cohort were male, and roughly two-thirds had type 3 disease. Patients received treatment 2 to 3 times weekly at 30 to 50 IU/kg over 12 months.

The annual bleeding rate during prophylaxis was low, at 4.6 ± 6.1, and 98.2% of the 56 bleeding episodes that did occur were minor. Among 45 treated episodes, 95.6% resolved with a single infusion, and no thrombotic events or factor VIII accumulation were observed. Three children were excluded from the per-protocol analysis, 2 for dosing deviations and 1 who discontinued after an adverse event not considered treatment-related.

"Prior research into prophylactic treatment for VWD has been very limited in this age group," said Akshat Jain, MD, MPH, principal investigator for WIL-33, in a statement. "We now have the pharmacokinetic data and insights to confirm that VWF prophylaxis is safe, effective and well-tolerated in pediatric patients with VWD, including children younger than 6 years of age.”

Findings from WIL-33 build on the earlier WIL-31 study, the largest prospective study of the prophylaxis treatment in patients 6 years and older, and show a similar prophylactic effect in the younger cohort. The treatment previously received orphan drug designation for routine prophylaxis in patients 6 and older, granting Octapharma up to 7 years of market exclusivity from its December 2023 approval date.

The approval closes a long-standing treatment gap: until now, clinicians managing children with severe, early-onset VWD had no FDA-sanctioned prophylactic option and relied on off-label or on-demand approaches. Recombinant VWF has separately demonstrated strong perioperative bleeding control in children with VWD, underscoring growing clinical confidence in VWF replacement across pediatric surgical and prophylactic settings.²

For payers, the FDA expansion adds a new indication to review for formulary and prior authorization criteria specific to children under 6, a population where treatment options, and therefore comparative cost and utilization data, have historically been sparse. As real-world use in this age group grows, health plans can expect increased attention to dosing frequency, infusion burden on caregivers, and long-term bleeding outcomes as points of comparison against on-demand management.

References

1. Octapharma USA announces expanded FDA approval of wilate for von Willebrand Disease prophylaxis in children younger than 6 years. Octapharma. News release. July 8, 2026. Accessed July 8, 2026. https://www.prnewswire.com/news-releases/octapharma-usa-announces-expanded-fda-approval-of-wilate-for-von-willebrand-disease-prophylaxis-in-children-younger-than-6-years-302820881.html
2. Bergmann S, Steinzor P. rVWF safely, effectively manages perioperative bleeding in pediatric patients: Shayla Bergmann, MD. AJMC^®. December 8, 2025. Accessed July 8, 2026. https://www.ajmc.com/view/rvwf-safely-effectively-manages-perioperative-bleeding-in-pediatric-patients-shayla-bergmann-md