Wilate (human factor VIII/von Willebrand factor) / Octapharma - LARVOL DELTA

Real-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study. (PubMed, J Comp Eff Res) - " Among 2540 on-demand RT users, WILFACTIN® was the main RT used, followed by VONCENTO®, VEYVONDI®, EQWILATE® and WILSTART®. This first real-world study suggests that VEYVONDI® seems to be a cost-saving RT compared with other RT. Future studies including clinical data should provide further evidence."

Journal • Hemophilia

Safety and Efficacy of pdVWF/FVIII 1:1 Prophylaxis in Children Under 6 Years with Severe VWD: Results from the WIL-33 Study (THSNA 2026) - P3 | "The efficacy and safety of prophylaxis with a plasma-derived (pd)VWF/factor VIII (FVIII) concentrate in a 1:1 activity ratio (pdVWF/FVIII 1:1, wilate®) was demonstrated in children (≥6 years) and adults in WIL-31... WIL-33 demonstrated that pdVWF/FVIII 1:1 prophylaxis was effective and well tolerated in children aged <6 years with severe VWD. WIL-33 is the first prospective trial specifically investigating prophylaxis in children and supports early, sustained prophylaxis in severe pediatric VWD. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • Allergic Rhinitis • Hemophilia • Immunology • Inflammation • IL33

Investigating the Effect of Population Pharmacokinetic-Guided pdVWF/FVIII (1:1) Prophylaxis for Severe VWD in a Real-World Setting: The PopPK-WILPROPHY Study (THSNA 2026) - "The efficacy and safety of prophylaxis with a plasma-derived VWF/factor VIII concentrate (pdVWF/FVIII 1:1, wilate®) has been demonstrated across ages and VWD types... The PopPK-WILPROPHY study will provide important evidence on the clinical utility of PopPK-guided prophylaxis with pdVWF/FVIII 1:1, supporting future implementation of personalized treatment strategies for individuals with severe VWD. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • PK/PD data • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

Comparative Clinical and Economic Evaluation of On-Demand versus Prophylactic Treatment with pdVWF/FVIII 1:1 and Recombinant VWF in Adults with Type 3 von Willebrand Disease in the United States (THSNA 2026) - P3 | "In the United States, plasma-derived VWF/FVIII in a 1:1 activity ratio (pdVWF/FVIII 1:1; wilate®) and recombinant VWF (rVWF; Vonvendi®) are the only concentrates approved for prophylaxis. wilate prophylaxis provides robust clinical benefits and significant cost advantages compared with OD therapy and rVWF prophylaxis in adults with severe type 3 VWD. These findings, supported by prospective intrapatient data and current U.S. pricing, indicate that wilate is a cost-efficient, first-line prophylactic option from both payer and societal perspectives in the US. Future analyses incorporating quality-adjusted life years and indirect health outcomes (e.g., joint preservation, chronic anemia prevention) will further define the long-term economic and therapeutic value of prophylaxis in this population."

Clinical • HEOR • Hematological Disorders • Hemophilia • IFNL1

Factor VIII and von Willebrand factor activity levels during long-term prophylaxis with wilate-Analyses from the WIL-31 study. (PubMed, Res Pract Thromb Haemost) - "During 12 months of wilate prophylaxis, there was no accumulation of FVIII or VWF regardless of age and VWD type, and no thrombotic events were reported. These findings from WIL-31 confirm and extend wilate's existing safety data."

Journal • Cardiovascular • Hematological Disorders • Hemophilia • Thrombosis

Efficacy and Safety of Prophylaxis With a Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Wilate) in Patients With Type 3 von Willebrand Disease-A WIL-31 Study Sub-Analysis. (PubMed, Eur J Haematol) - P3 | "Prophylaxis with wilate was effective and well tolerated in patients with type 3 VWD, in all age groups."

Journal • Hemophilia

Comparative analysis of prophylaxis with 1:1 ratio pdVWF/FVIII concentrate vs. recombinant VWF in type 3 von willebrand disease adults in the United States: Clinical and economic perspective (ASH 2025) - P3 | "In the United States (U.S.), plasma-derived VWF/FVIII (pdVWF/FVIII; wilate®) and recombinant VWF (rVWF; Vonvendi®) are the only two VWF concentrates approved forroutine prophylaxis in VWD. Based on current prophylaxis regimens and U.S. pricing, pdVWF/FVIIIdemonstrates favorable economic value. These findings support its consideration as a cost-saving, first-line prophylactic option in this population."

Clinical • Hemophilia

Real-world study of population pharmacokinetic-guided pdVWF/FVIII (1:1) prophylaxis in people with severe von Willebrand disease: The PopPK-WILPROPHY study (ASH 2025) - "While the efficacy and safety ofprophylaxis with a plasma-derived VWF/factor VIII concentrate (pdVWF/FVIII) in a 1:1 activity ratio(wilate®) has been demonstrated in adults, adolescents and children with severe VWD prophylaxis, dataon pharmacokinetic (PK)-guided prophylaxis in persons with VWD are lacking and individualizedprophylaxis has not yet been implemented or operationalized.Aims: To investigate the impact of PK-guided prophylaxis with a 1:1 pdVWF/FVIII concentrate on clinicaloutcomes in people with clinically severe VWD during routine clinical practice. The PopPK-WILPROPHY study will be a prospective, observational, single-arm, multicenterstudy enrolling 70 patients with clinically severe VWD... This study is planned to start in Q1 2026 and aims to enroll 70 patients at approximately 20centers in the USA, Canada, Europe, Asia and Latin America.Conclusion(s): The PopPK-WILPROPHY study will provide important evidence on the clinical utility ofPopPK-guided 1:1..."

Clinical • PK/PD data • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

Plasma-derived VWF/FVIII prophylaxis in children under 6 with VWD: First results from wil-33 (ASH 2025) - P3 | "Efficacy and safety of prophylaxis with a plasma-derived (pd)VWF/factor VIII (FVIII)concentrate in a 1:1 activity ratio (wilate®) has been demonstrated in WIL-31, the largest study in VWD todate... A total of 12 patients were enrolled and treated in this global study. At baseline the median(range) age was 2.0 (1.0–5.0), there were 6 male patients (50%), 4 patients (33.3%) had VWD type 2, and 8patients (66.7%) had VWD type 3. The per-protocol robustness (PPR) set, a subset of the full analysis set(FAS), excluded three patients with deviations mainly from the recommended dosing."

Clinical • Allergic Rhinitis • Hemophilia • Immunology • Inflammation • IL33

Real-world treatment characteristics and outcomes in patients with von Willebrand disease treated prophylactically with plasma-derived or recombinant von Willebrand factor in europe: A multi-center, retrospective study (ASH 2025) - "Of the 11 patients who received continuous pdVWF prophylaxis, 7 (63.6%)received Wilfactin/Willfact, 3 (27.3%) received Voncento, and 1 (9.1%) received Wilate/Eqwilate...During the post-index period (median duration 66.0 months), themedian (range) ABR was 0.4 (0.0–29.3).ConclusionsThis real-world study investigating the use of rVWF and pdVWF prophylaxis in clinical practice in Europehighlights the effectiveness of rVWF prophylaxis in patients with VWD. Further research involving a largersample of patients is warranted to enable comparisons between rVWF and pdVWF prophylaxis."

HEOR • Real-world • Real-world evidence • Retrospective data • Gynecology • Hematological Disorders • Hemophilia • Rheumatology

Pharmacokinetic-pharmacodynamic modelling of von willebrand factor/factor VIII prophylaxis in von willebrand disease (ASH 2025) - P3 | "Effective clinical implementationof PK-guided dosing requires knowledge of both the interpatient PK variability and the relationshipbetween drug exposure and bleeding risk.Objective:To develop a population PK model for the plasma-derived VWF/factor VIII (FVIII) 1:1 concentrate (Wilate®)in patients with severe VWD and to explore the relationship between VWF/FVIII activity and bleedingevents during prophylaxis.A nonlinear mixed-effects PK modelling approach was applied to VWF:ristocetin cofactor (VWF:RCo) andFVIII activity (FVIII:C) data from pediatric and adult patients participating in a prospective single-dose (WIL-12) and a 1-year prospective, prophylaxis phase 3 trial (WIL-31; NCT04052698)...In this study, we developed a population PK model for the plasma-derived VWF/FVIII 1:1 concentrate thatcan support clinicians in optimising VWD therapy by assessment of exposure PK metrics (trough levels,area under the curve, time above a defined threshold) and VWF:RCo..."

PK/PD data • Hemophilia

Cost comparison of prophylactic vs. on-demand use of a 1:1 ratio pdVWF/FVIII concentrate in type 3 von willebrand disease with severe bleeding phenotype in the United States: A payer and societal perspective (ASH 2025) - "This studypresents a cost comparison of Wilate® prophylaxis versus OD treatment in patients with Type 3 VWD anda severe bleeding phenotype, using real-world prospective data from the WIL-29 and WIL-31 phase 3studies...These findings support the expanded use of VWF concentrate prophylaxis withincomprehensive VWD management strategies. Future research incorporating broader direct/indirectcosts, including those related to long-term health outcomes, e.g., joint deterioration, chronic anaemia,heavy menstrual bleeds, mental health and quality-adjusted life years (QALYs), may further strengthenthe economic and therapeutic value of prophylactic treatment."

HEOR • Reimbursement • US reimbursement • Hematological Disorders • Hemophilia • IFNL1

Individualizing dosing frequency may improve the efficacy of prophylaxis in patients with von Willebrand disease-a WIL-31 subanalysis. (PubMed, Res Pract Thromb Haemost) - P3 | "The efficacy and safety of prophylaxis with wilate, a plasma-derived von Willebrand factor/factor VIII concentrate, was demonstrated in patients with von Willebrand disease of all types in the WIL-31 study...In most cases, there were underlying medical conditions that necessitated the increased dosing frequency, indicating that personalization of prophylaxis may be beneficial in patients with von Willebrand disease. NCT04052698; https://clinicaltrials.gov/study/NCT04052698."

Journal • Dental Disorders • Hemophilia • Rheumatology

Effect of Factor VIII and Von Willebrand Factor Activities on Number of Bleeding Events in Previously Treated Patients with Von Willebrand Disease on Regular Prophylaxis with a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate (ASH 2023) - P3 | "The WIL-31 study demonstrated the efficacy of prophylaxis with a plasma-derived VWF/factor VIII concentrate containing VWF and FVIII in a 1: 1 activity ratio (pdVWF/FVIII; wilate ®) in adults and children with VWD... No accumulation of FVIII or VWF in the plasma was reported in 33 patients who received regular prophylaxis with pdVWF/FVIII for 12 months, regardless of VWD type and age."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Thrombosis • IFNL1

Efficacy of Regular Prophylaxis with a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate in Reducing Nose Bleeds in Children and Adults with Von Willebrand Disease (ASH 2023) - P3 | "The WIL-31 study demonstrated the efficacy of prophylaxis with a plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrate containing VWF and FVIII in a 1: 1 activity ratio (wilate ®) in adults and children with VWD of all types...1% (62/72) requiring only a single infusion; two required concomitant treatment with tranexamic acid. The data indicate thatprophylaxis with pdVWF/FVIII was effective at reducing nose bleeds in patients with VWD type 2 and 3, and across all age groups."

Clinical • Hemophilia • IFNL1

Immunogenicity of a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate during the Wil-31 Prophylaxis Study in Patients with Von Willebrand Disease (ASH 2023) - P3 | "The WIL-31 study demonstrated the efficacy of prophylaxis with a plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrate containing VWF and FVIII in a 1: 1 activity ratio (wilate ®) in adults and children with VWD of all types... VWF inhibitors were detected in 2 of the 33 patients on prophylaxis with pdVWF/FVIII in the WIL-31 study and subsequent investigation revealed that both patients were already positive for VWF inhibitors prior to the study start. In one of the patients, the inhibitor resolved during continued prophylaxis. Despite the inhibitors, prophylaxis was effective in both patients with an ≥85% reduction in ABR compared to on demand treatment."

Clinical • Hemophilia • IFNL1

Efficacy of Regular Prophylaxis with a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate in Reducing Heavy Menstrual Bleeding in Females with Von Willebrand Disease (ASH 2023) - P3 | "A recent prospective study found that recombinant VWF was not superior to tranexamic acid at reducing HMB in women with VWD. The WIL-31 study demonstrated the efficacy of prophylaxis with a plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrate containing VWF and FVIII in a 1:1 activity ratio (wilate®) in adults and children with VWD of all types... Prophylaxis with pdVWF/FVIII was efficacious in reducing HMB compared with on-demand treatment in 7 female patients with VWD. None of the cases of HMB during prophylaxis was severe enough to require additional treatment. Among women with VWD, HMB remains a major burden and further research is required."

Clinical • Hemophilia • Long-acting Reversible Contraceptives • IFNL1

Efficacy and Safety of Prophylaxis with a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate in Children and Adolescents with Von Willebrand Disease – a Sub-Analysis of Data from the Wil-31 Study (ASH 2023) - P3 | "The WIL-31 study demonstrated the efficacy of prophylaxis with a plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrate containing VWF and FVIII in a 1:1 activity ratio (wilate®) in adults and children with VWD of all types... Prophylaxis with pdVWF/FVIII was efficacious and well tolerated in children and adolescents with all types of VWD."

Clinical • Hemophilia • IFNL1

FVIII and VWF PK: Key Aspects in Safety and Efficiency of VWF Concentrates. Review and Reflection (ASH 2024) - P3 | "These VWFCs are of plasma origin, with one recombinant origin, rVWFC (Vonvendi®), containing only VWF. Among the plasma-derived ones, we have plasma derived Von Willebrand Factor Concentrate with FVIII (pd-VWFC) (Wilate®) with a VWF/FVIII ratio of 1 : 1, and other pd-VWFC with a low FVIII content (Wilfact®)(FVIII : VWF ratio <0.2 : 1).Methods : For this analysis, data for rVWFC comes from post hoc analysis of prospective clinical trial (CT) NCT02973087, 2 and for pd-VWFC (with FVIII), from prospective CT NCT04052698...4Regarding this risk, it could be assumed that VWFCs without FVIII would be exempt from it, but TEs were reported in scheduled surgeries with rVWFC 5 and with other VWFCs/FVIII with a lower proportion of FVIII than the pd-VWFC with FVIII analized (for example, pd-VWFC rich in VWF, Haemate P®)...5,13 With pd-VWFC (ratio VWF/FVIII 1 : 1), the obtained FVIII peak is higher, coinciding with the VWF peak, generating a higher thrombin..."

Clinical • Review • Cardiovascular • Hematological Disorders • Hemophilia • Thrombosis

Increased Prophylactic Dosing Frequency Effectively Improve Bleeding Rates in Von Willebrand Disease Patients Who Require Higher Hemostatic Coverage - a Sub-Analysis of Data from the Wil-31 Study (ASH 2024) - P, P3 | "The results of the WIL-31 study showed that prophylaxis with a plasma-derived VWF/factor VIII (FVIII) concentrate containing VWF and FVIII in a 1 : 1 activity ratio (pdVWF/FVIII; wilate®) was highly efficacious in adults and children, males and females, with VWD of all types.Aims : To investigate bleeding outcomes in VWD patients in whom the dosing frequency was increased from 2 to 3 infusions weekly during the WIL-31 study.Methods : WIL-31 was a prospective, non-controlled, international, multicenter Phase 3 trial that enrolled male and female patients, aged ≥6 years with VWD type 1 (VWF : RCo <30 IU/dL), type 2 (except 2N), or type 3...Of those who required an increase in the dosing frequency, most had underlying circumstances that precipitated the change. Patients have diverse needs that must be considered when deciding on the dosing regimen for an individual."

Clinical • Allergic Rhinitis • Dental Disorders • Hemophilia • Immunology • Inflammation • Rheumatology • IFNL1

Efficacy and Safety of Prophylaxis with a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate Among Von Willebrand Disease Patients across All ABO Antigen Blood Groups (ASH 2024) - P, P3 | "No serious adverse events related to study treatment and no thrombotic events were observed. Conclusion : wilate® prophylaxis was efficacious at reducing bleeding in patients with VWD regardless of ABO blood group."

Clinical • Hemophilia • IFNL1

Disparities in Real-World Treatment Patterns Among Patients with Von Willebrand Disease in a Large US Population-Based Dataset (ASH 2024) - "On-demand medications included antifibrinolytics and desmopressin, which were used 46% and 31% of the time, respectively, while 23% used VWF replacement therapies. Among prophylaxis users, 49% of patients were treated with VWF replacement therapies, most commonly Humate-P (77%), followed by wilate (16%), Vonvendi (11%), and Alphanate (8%)...Hemlibra was used in 2% of patients, mainly to treat those with joint bleeds...While heavy menstrual bleeding and nosebleeds were the most common bleeding phenotypes among VWD patients with bleeding comorbidities (and likely underrepresented in claims data), only a minority of patients received VWF replacement therapies. These findings suggest a high unmet need for better treatment options for VWD patients who may benefit from prophylactic therapy."

Clinical • HEOR • Real-world • Real-world evidence • Anemia • Gastrointestinal Disorder • Hematological Disorders • Hemophilia

A Budget Impact Analysis of Switching From Von Willebrand Factor/Factor VIII Plasma-Derived Concentrate With 2.4-1 Ratio to the Concentrate With 1-1 Ratio for the Management of Von Willebrand Disease in Qatar (ISPOR-EU 2025) - "OBJECTIVES: To assess the financial impact of introducing a plasma-derived (pd) von Willebrand Factor (VWF)/ Factor VIII (FVIII) concentrate with a 1-1 activity ratio (Wilate®), for von Willebrand Disease management (vWD) in Qatar... In Qatar, using pdVWF/FVIII concentrate with a 1-1 ratio for the management of vWD would lead to significant cost savings over the five-year time horizon. These findings and existing clinical evidence support the introduction of the new pdVWF/FVIII concentrate with 1-1 ratio into treatment protocols."

HEOR • Hemophilia

Budget impact analysis of Haemate-P as long-term prophylaxis and on-demand therapy for von Willebrand disease in Spain. (PubMed, J Med Econ) - "In univariate and multiway deterministic sensitivity analyses, the alternative scenario remained cost-saving. Haemate-P was found to be a cost-saving strategy, and increased use of Haemate-P over Fanhdi, and Wilate is expected to reduce overall costs to the healthcare system in Spain."

HEOR • Journal • Hemophilia

von Willebrand Factor (VWF) Inhibitors in Two Brothers with von Willebrand Disease: A Case Report. (PubMed, TH Open) - "Here, we report two brothers with type 3 VWD who at the 6-month visit were found to have VWF inhibitors, which on further investigation were found to have already been present before the study. Despite the presence of inhibitors, neither patient showed any clinical symptoms, and prophylaxis with wilate led to a ≥85% reduction in ABR in both boys compared with on-demand treatment."

Journal • Hemophilia