FDA Expands Wilate Approval for Routine Prophylaxis in Children Younger Than 6 With von Willebrand Disease

The FDA has expanded the approval of Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]; Octapharma) for routine prophylaxis to reduce the frequency of bleeding episodes in children aged younger than 6 years with von Willebrand disease (VWD).¹

The approval extends the prophylactic indication for Wilate to a younger pediatric population with severe VWD. Wilate is now indicated in adult and pediatric patients with VWD for on-demand treatment and control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis to reduce bleeding frequency.²

VWD is an inherited bleeding disorder caused by quantitative or qualitative abnormalities in von Willebrand factor, a protein involved in platelet adhesion and factor VIII stabilization. Patients with VWD may experience mucocutaneous bleeding, prolonged bleeding after injury or surgery, heavy menstrual bleeding, epistaxis, gastrointestinal bleeding, or joint and muscle bleeding, depending on disease type and severity.³

Evidence-based guidelines from the American Society of Hematology, International Society on Thrombosis and Haemostasis, National Hemophilia Foundation, and World Federation of Hemophilia recommend long-term prophylaxis for patients with VWD who have a history of severe and frequent bleeding. This expanded approval provides an FDA-approved prophylactic option for patients aged younger than 6 years, a population in which prospective prophylaxis data have standardly been limited.⁴

WIL-33 Trial Supports Expanded Approval

The expanded indication is supported by data from WIL-33 (NCT04953884), an open-label, prospective, noncontrolled, international, multicenter phase 3 trial evaluating the efficacy, immunogenicity, pharmacokinetics, and safety of Wilate prophylaxis in children younger than 6 years with severe VWD.^1,5

The study enrolled and treated 12 pediatric patients aged younger than 6 years with severe VWD, defined as von Willebrand factor ristocetin cofactor activity of less than 20%. At baseline, the median patient age was 2.0 years, with a range of 1.0 to 5.0 years. Six patients were male, 4 patients had type 2 VWD, and 8 patients had type 3 VWD.¹

Patients received Wilate 2 to 3 times weekly for 12 months at a recommended dose of 30 to 50 IU/kg. The primary end point was total annualized bleeding rate during prophylaxis, with safety and tolerability assessed throughout the study.¹

During 12 months of prophylaxis, the annualized bleeding rate was 4.6 ± 6.1. Among 56 bleeding episodes reported during the study, 98.2% were classified as minor. Of the 45 bleeding episodes that were treated, 95.6% required only 1 infusion.¹

According to the prescribing information, the total annualized bleeding rate for routine prophylaxis was 4.6 in patients younger than 6 years, compared with 3.73 in patients aged 6 to 12 years and 4.28 in patients aged 12 to younger than 17 years.²

Safety and Tolerability

Prophylaxis with Wilate was well tolerated in the WIL-33 population. No thrombotic events or factor VIII accumulation were observed during the trial.¹

Wilate is contraindicated in patients with known hypersensitivity reactions, including anaphylactic or severe systemic reactions, to human plasma-derived products, any ingredient in the formulation, or components of the container.²

Anaphylaxis and severe hypersensitivity reactions may occur with Wilate. Thromboembolic events are also possible, and clinicians should monitor plasma factor VIII activity levels. Neutralizing antibodies to von Willebrand factor and factor VIII have occurred following administration of Wilate, and testing is recommended if plasma von Willebrand factor or factor VIII levels fail to increase as expected or if bleeding is not controlled after administration.²

Because Wilate is made from human plasma, it carries a risk of transmitting infectious agents.²

Implications for Pediatric VWD Management

The expanded approval may help address an unmet need in the prophylactic management of very young children with severe VWD who experience frequent or clinically significant bleeding episodes. Prior to this approval, Wilate was approved for routine prophylaxis in adults and children aged 6 years and older with VWD; the new indication extends prophylactic use to children younger than 6 years.¹

The WIL-33 findings also build on data from WIL-31, a prospective study evaluating Wilate prophylaxis in adults and children aged 6 years and older with VWD. Together, the data support the use of Wilate prophylaxis across pediatric age groups, including patients with severe VWD who may require ongoing bleed prevention.¹

For pharmacists and hematology care teams, the updated indication provides an FDA-approved routine prophylaxis option for younger pediatric patients with VWD. The recommended routine prophylaxis dose for patients younger than 6 years is 30 to 50 IU/kg administered 2 or 3 times weekly. For patients aged 6 years and older with VWD, the recommended routine prophylaxis dose is 20 to 40 IU/kg administered 2 or 3 times weekly.²

REFERENCES

1. Octapharma USA. Octapharma USA announces expanded FDA approval of wilate® for von Willebrand disease prophylaxis in children younger than 6 years. News Release. Released July 8, 2026. Accessed July 9, 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. Wilate® [prescribing information]. Octapharma USA; 2026. https://www.octapharmausa.com/products/product-overview/wilate

3. Sholzberg M, Weyand AC, Lai JS, et al. Diagnosis and disease burden of von Willebrand disease in a large US population-based dataset. Blood. 2024;144:3980-3981. doi:10.1182/blood-2024-206631

4. Connell NT, Flood VH, Brignardello-Petersen R, et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Adv. 2021;5(1):301-325. doi:10.1182/bloodadvances.2020003264

5. ClinicalTrials.gov. Efficacy, PK, immunogenicity, and safety of wilate as routine prophylaxis in VWD patients under 6 years of age. NCT04953884. Accessed July 9, 2026. https://clinicaltrials.gov/study/NCT04953884

6. Akshat Jain, Cindy Leissinger, Vladimir Vdovin, Pavel Zharkov, et al. Plasma-derived VWF/FVIII prophylaxis in children under 6 with VWD: First results from wil-33. Blood 2025; 146 (Supplement 1): 4855. doi:10.1182/blood-2025-4855