Octapharma USA Announces Expanded FDA Approval of wilate® for von Willebrand Disease Prophylaxis in Children Younger Than 6 Years

PR Newswire

PARAMUS, N.J., July 8, 2026

PARAMUS, N.J., July 8, 2026 /PRNewswire/ — On July 2, 2026, the U.S. Food and Drug Administration (FDA) approved the use of wilate^® (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) for routine prophylaxis to reduce the frequency of bleeding episodes in pediatric patients with von Willebrand Disease (VWD) who are younger than 6 years of age.ⁱ This approval expands access to the first von Willebrand Factor (VWF) concentrate indicated for prophylactic treatment across all ages and forms of VWD, the most common inherited bleeding disorder.ⁱⁱ

The expanded label is based on data from the Phase 3 WIL-33 clinical trial (ClinicalTrials.gov ID: NCT04953884), an international study to research the safety and efficacy of VWF prophylaxis in children with VWD under the age of 6.

“Evidence-based treatment guidelines recommend prophylactic treatment for VWD patients with a history of severe, frequent bleeds,ⁱⁱⁱ” said Flemming Nielsen, President, Octapharma USA. “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.”

The open-label WIL-33 trial enrolled and treated 12 patients, all under the age of 6 and diagnosed with severe VWD. Wilate^® was administered two to three times per week at a recommended dose of 30 to 50 international units (IU)/kg over 12 months. The primary endpoint was the total annualized bleeding rate (TABR) during prophylaxis, with safety and tolerability assessed throughout the study.

Researchers concluded that wilate^® was efficacious in the prevention and treatment of bleeding episodes in children under age 6, across all VWD types. The annual bleeding rate (ABR) measured during 12 months of prophylaxis was low (4.6 ± 6.1). Of the 56 bleeding episodes that occurred, 98.2% were determined to be minor in nature. In terms of treatment efficacy, of the 45 bleeding episodes treated, 95.6% required only one infusion.^iv

The WIL-33 findings complement results from the WIL-31 study, the largest prospective study to date evaluating prophylaxis with wilate^® in adults and children aged 6 years and older with VWD. WIL-33 revealed a similar prophylactic effect in younger children to the efficacy demonstrated in WIL-31.ⁱ

Prophylaxis with wilate^® was well tolerated in this population, with no thrombotic events or FVIII accumulation observed over the course of the trial.^iv

“Prior research into prophylactic treatment for VWD has been very limited in this age group,” said Akshat Jain, MD, MPH, principal investigator for the WIL-33 trial. “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.”

The FDA previously granted wilate^® with an orphan drug designation for routine prophylaxis to reduce the frequency of bleeding episodes in adults and children 6 years of age and older with VWD, granting Octapharma a potential seven years of market exclusivity from the date of approval (December 1, 2023).

About the study
WIL-33 was an open-label, prospective, non-controlled, international, multicenter phase 3 clinical trial investigating the efficacy, immunogenicity, and safety of wilate^® prophylaxis and pharmacokinetics in 12 pediatric patients under 6 years old with severe VWD (VWF ristocetin cofactor activity [VWF:RCo] <20%). At baseline the median age was 2.0 (1.0–5.0), there were six male patients (50%), four patients (33.3%) had VWD type 2, and eight patients (66.7%) had VWD type 3.

Three patients were excluded from the per-protocol robustness (PPR) set, with two participants removed due to deviations from the recommended prophylactic dose; one additional patient prematurely discontinued treatment due to adverse events that were not considered treatment-emergent.

About wilate^®Wilate^® (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) Lyophilized Powder for Solution for Intravenous Injection, is indicated in adult and pediatric patients with von Willebrand disease (VWD) for:

On-demand treatment and control of bleeding episodes.
Perioperative management of bleeding.
Routine prophylaxis to reduce the frequency of bleeding episodes.

Wilate^® is indicated in adult and pediatric patients 12 years of age and older with hemophilia A for:

On-demand treatment and control of bleeding episodes.
Routine prophylaxis to reduce the frequency of bleeding episodes.

Important Safety Information
CONTRAINDICATIONS
Do not use 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.

WARNINGS AND PRECAUTIONS
Anaphylaxis and severe hypersensitivity reactions are possible. Thromboembolic events may occur. Monitor plasma levels of FVIII activity. Neutralizing antibodies (inhibitors) to VWF and FVIII have occurred following administration of wilate^®. Test for neutralizing antibodies if plasma VWF and/or FVIII level fail to increase as expected or if bleeding is not controlled after wilate^® administration. Wilate^® is made from human plasma and carries the risk of transmitting infectious agents.

For full prescribing information, please visit wilateusa.com/pi.

About Octapharma
Octapharma is one of the world’s largest human protein manufacturers, developing and producing therapies from human plasma and human cell lines. Headquartered in Lachen, Switzerland, Octapharma has over 11,000 employees and serves patients in 117 countries across Immunotherapy, Hematology, and Critical Care.

With seven R&D sites, five manufacturing facilities in Europe, and more than 200 plasma donation centers in the U.S. and Europe, Octapharma has delivered trusted therapies for over 40 years. The company is committed to improving lives through innovation, quality, and a deep focus on patient care and clinical partnership. To learn more please visit www.octapharma.com.

References

ⁱ Wilate^® Prescribing Information
ⁱⁱ 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. https://doi.org/10.1182/blood-2024-206631
ⁱⁱⁱ Connell NT, Flood VH, Brignardello-Peterson R, et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Adv. 2021; 5(1):301-325. https://doi.org/10.1182/bloodadvances.2020003264
^iv Jain A, Leissinger C, Vdovin V, et al. Plasma-Derived VWF/FVIII Prophylaxis in Children Under 6 With VWD: First Results From WIL-33 [Poster abstract]. Blood. 2025; 146:4855-4856. https://doi.org/10.1182/blood-2025-4855