FDA expands wilate approval to routine prophylaxis for all types of von Willebrand disease

The FDA has expanded the approved label for wilate (von Willebrand factor/coagulation factor VIII complex [human]) to include routine prophylaxis for adults and children aged 6 years and older with any type of von Willebrand disease (VWD), making it the first VWF concentrate approved for prophylactic use across all VWD subtypes.¹ The decision, announced by manufacturer Octapharma USA on December 5, 2023, was supported by phase 3 data showing an 84% reduction in mean total annualized bleeding rate (ABR) when patients with severe VWD were switched from on-demand treatment to routine prophylaxis.^1,2

“Long-term prophylaxis with VWF concentrate, as compared to on-demand treatment for bleeding, is recommended for patients with severe VWD,” said Shveta Gupta, MD, a pediatric hematologist-oncologist at the Haley Center for Children’s Cancer and Blood Disorders at Orlando Health Arnold Palmer Hospital for Children.¹ “The approval of wilate for VWD prophylaxis is a welcome new treatment option that can be life-saving for many patients.”¹

WIL-31 trial design and bleeding-rate outcomes in severe VWD

The label expansion rests on WIL-31, a prospective, noncontrolled, international, multicenter phase 3 trial of wilate prophylaxis over 12 months in patients aged 6 and older with severe VWD of any type.¹ Enrollment required prior participation in WIL-29, a 6-month observational study of on-demand wilate treatment, and at least 6 qualifying bleeding episodes (excluding menstrual bleeding), with at least 2 treated with a VWF-containing product.¹ Patients received prophylactic wilate at 20 to 40 IU/kg 2 to 3 times weekly for 12 months.¹ In the published cohort (n = 33; median age, 18 years), mean total ABR during prophylaxis was 5.2, an 84.4% reduction from the on-demand period, meeting the trial’s primary endpoint of a greater than 50% reduction.² Mean spontaneous ABR fell 87%, and 30.3% of patients had zero bleeding events during prophylaxis.² No treatment-related serious adverse events or thrombotic events were reported.^1,2

Disease burden and unmet need in von Willebrand disease

VWD is the most common inherited bleeding disorder in the United States, affecting up to 1% of the population, or roughly 3.3 million people.¹ It occurs at similar rates in men and women but is more often recognized in women because of heavy menstrual bleeding or postpartum hemorrhage.¹ The disease spans 3 major subtypes—type 1, type 2, and type 3, the most severe—and until this approval, patients with non–type 3 VWD had no FDA-approved VWF concentrate specifically indicated for routine prophylaxis, leaving many managed with on-demand treatment despite recurrent bleeding.¹

Wilate mechanism and prior regulatory history

Wilate is a plasma-derived concentrate containing VWF and factor VIII in a near-physiologic 1:1 ratio, administered intravenously.¹ Before this expansion, its VWD indication was limited to on-demand treatment and perioperative bleeding management; the product also carries an indication for prophylaxis and on-demand treatment in adolescents and adults with hemophilia A.¹ Boxed warnings and precautions include anaphylaxis and hypersensitivity reactions, thromboembolic events, the need to monitor factor VIII activity, and the risk of neutralizing antibody development, particularly in type 3 VWD.¹

Interpreting the evidence and remaining questions

WIL-31’s design—comparing prophylaxis to each patient’s own prior on-demand bleeding history rather than to a concurrent randomized control—limits causal certainty and leaves open the possibility that regression to the mean or closer trial monitoring contributed to the observed bleeding reduction.^1,2 The cohort was also small, and subgroup data in children, adolescents, and type 3 VWD patients derive from post hoc analyses rather than prespecified comparisons.² Octapharma USA President Flemming Nielsen framed the approval as addressing a long-standing treatment gap, stating that patients “have been forced to live with far too many bleeding episodes while receiving on-demand treatment.”¹ Longer-term registry data and real-world dosing-frequency studies, some already underway, will help clarify durability of response and optimal dosing intervals outside the trial setting.

References

1. Octapharma USA. FDA grants expanded approval to wilate® as the first VWF concentrate for prophylaxis in all types of VWD. Published December 5, 2023. Accessed July 8, 2026. https://www.octapharmausa.com/news/press-release/2023/octapharma-usa-fda-grants-expanded-approval-to-wilate-as-the-first-vwf-concentrate-for-prophylaxis-in-all-types-of-vwd

2. Djambas Khayat C, Sidonio RF Jr, Boban A, et al. Von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease. Blood Adv. 2024;8(6):1405-1414. doi:10.1182/bloodadvances.2023011671

3. US Food and Drug Administration. WILATE (von Willebrand factor/coagulation factor VIII complex [human]) approved product information. Accessed July 8, 2026. https://www.fda.gov/vaccines-blood-biologics/approved-blood-products/wilate

4. Centers for Disease Control and Prevention. What is von Willebrand disease? Accessed July 8, 2026. https://www.cdc.gov/von-willebrand/