FDA Approves Expanded Use of Wilate for Von Willebrand Disease Prophylaxis in Children Under Age 6

Octapharma USA announced that the FDA has approved an expanded indication for Wilate (von Willebrand Factor/Coagulation Factor VIII Complex, Human) to include routine prophylaxis to reduce the frequency of bleeding episodes in children younger than 6 years with any type of von Willebrand disease (VWD).¹ The approval, granted on July 2, 2026, marks the first FDA-approved option for routine prophylaxis specifically in this youngest pediatric age group, filling a longstanding clinical gap in the management of the most common inherited bleeding disorder.¹ The action reflects a growing regulatory focus on extending approved biologic therapies to underserved pediatric populations in rare hematologic diseases, where treatment options have historically been extrapolated from adult data rather than established through dedicated pediatric trials.

What is von Willebrand disease and what is the clinical significance of the under-6 population?

Von Willebrand disease is the most common inherited bleeding disorder worldwide, affecting an estimated 1% of the global population, though registry-reported prevalence averages only 25.6 per million — reflecting significant under diagnosis driven by clinical and laboratory heterogeneity, variable disease expression, and limited awareness among non-specialist healthcare providers.² VWD is caused by a deficiency or dysfunction of von Willebrand factor (VWF), a large multimeric glycoprotein that plays a dual role in hemostasis: facilitating platelet adhesion to damaged vessel walls and serving as a stabilizing carrier protein for coagulation factor VIII (FVIII).³

The disease encompasses three major subtypes — Type 1 (partial quantitative VWF deficiency), Type 2 (qualitative VWF abnormalities), and Type 3 (severe quantitative deficiency) — with a correspondingly broad spectrum of bleeding severity from mild mucocutaneous bleeding to life-threatening hemorrhage.² Children with severe VWD are at elevated risk of spontaneous and trauma-related bleeding from infancy, including joint bleeds, mucosal hemorrhage, and intracranial bleeding — risks that are particularly acute in the pre-school years when falls, injuries, and surgical procedures such as dental extractions and tonsillectomies are common.

Despite this vulnerability, pediatric hematologists managing children under 6 years with VWD have lacked an FDA-approved option for routine prophylaxis in this age group — relying instead on clinical practice guidelines and off-label use of adult-approved therapies. The expanded Wilate indication directly addresses this gap.

What did the Phase 3 WIL-33 Trial show?

The approval is supported by data from the Phase 3 WIL-33 clinical trial (NCT04953884), an international, multicenter, open-label study evaluating the safety and efficacy of Wilate prophylaxis in children with VWD under the age of 6.¹ The trial demonstrated that routine prophylaxis with Wilate significantly reduced the frequency of bleeding episodes in young pediatric patients, with a safety profile consistent with that established in older patients and adults.¹

Wilate was generally well-tolerated across the pediatric population. Prescribing information includes warnings for anaphylaxis and severe hypersensitivity reactions, thromboembolic events, monitoring of plasma FVIII activity levels, and the rare possibility of neutralizing antibody (inhibitor) development to VWF and FVIII — consistent with the class-level risk profile of plasma-derived VWF/FVIII concentrates.¹

"Evidence-based treatment guidelines recommend prophylactic treatment for VWD patients with a history of severe, frequent bleeds," said Flemming Nielsen, president of Octapharma USA. "Until now, pediatric hematologists have not had an FDA-approved option for routine prophylaxis in young children with VWD. This approval recognizes the importance of treating patients as young as possible, giving them the best chance to avoid joint damage and other complications of recurrent bleeding."¹

What is Wilate and how does it work?

Wilate is a plasma-derived, high-purity VWF/FVIII complex concentrate developed and manufactured by Octapharma, one of the world's largest human protein manufacturers.¹ The product contains both VWF and FVIII in a physiological ratio, closely mimicking the natural protein complex present in human plasma. It is produced from human plasma using a double virus inactivation process — solvent/detergent treatment and dry heat treatment — designed to minimize the risk of transmissible infectious agents.¹

Wilate is administered intravenously and is indicated for treatment and prophylaxis of bleeding in adults and children with VWD when desmopressin treatment is either ineffective or not indicated.¹ The product holds existing FDA approval for adults and pediatric patients aged 6 and older; today's expanded label extends coverage to the youngest pediatric population.

The plasma-derived VWF/FVIII class sits within the broader spectrum of biologic protein replacement therapies for inherited bleeding disorders — a field that has increasingly expanded into recombinant formats (such as vonicog alfa, a recombinant VWF concentrate approved for adults) and, in hemophilia, into extended half-life engineered biologics and non-replacement approaches such as emicizumab. For VWD specifically, plasma-derived VWF/FVIII concentrates remain the standard of care for patients requiring factor replacement, with recombinant options emerging but not yet covering the full range of VWD subtypes and age groups.⁴

What does this mean for pediatric VWD management?

The WIL-33 approval arrives in the context of growing recognition that VWD remains substantially underdiagnosed and undertreated, particularly in children and in female patients whose bleeding symptoms are frequently attributed to other causes.² Prophylactic treatment — rather than episodic on-demand therapy — is increasingly supported by hematology guidelines for patients with severe or frequently recurring disease, given evidence that early intervention can prevent the cumulative joint and organ damage associated with recurrent bleeding.

For pediatric hematologists managing the youngest patients with VWD, the approval of Wilate provides a regulatory framework for prophylaxis decisions that was previously absent, potentially facilitating formulary access, insurance coverage, and standardization of care in a population where clinical practice has varied widely across treatment centers.

Octapharma serves patients in 117 countries across its Immunotherapy, Hematology, and Critical Care divisions, with wilate among its core hematology portfolio.¹

"Until now, pediatric hematologists have not had an FDA-approved option for routine prophylaxis in young children with VWD. This approval recognizes the importance of treating patients as young as possible, giving them the best chance to avoid joint damage and other complications of recurrent bleeding."
— Flemming Nielsen, President, Octapharma USA¹

References

1. Octapharma USA Announces Expanded FDA Approval of wilate® for Von Willebrand Disease Prophylaxis in Children Younger Than 6 Years. (2026 Jul 8). PR Newswire. 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. Seidizadeh O, Abdul-Kadir R, Mannucci PM, Peyvandi F. (2026 Jan 20). Beyond a century of discovery: the global and persistent burden of underdiagnosis in von Willebrand disease. Res Pract Thromb Haemost. https://pmc.ncbi.nlm.nih.gov/articles/PMC12925064/
3. Leebeek FWG, Eikenboom JCJ. (2016 Nov 24). Von Willebrand's disease. N Engl J Med. https://pubmed.ncbi.nlm.nih.gov/27959741/
4. Barraclough A, Bär I, van Duijl T, et al. (2025 Sep 22). Rewriting the script: gene therapy and genome editing for von Willebrand disease. Front Genome Ed. https://pmc.ncbi.nlm.nih.gov/articles/PMC12497766/
5. Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age. ClinicalTrials.gov. https://clinicaltrials.gov/study/NCT04953884