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

Efficacy of regular prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate with a 1:1 activity ratio in reducing heavy menstrual bleeding in girls/women with von Willebrand disease. (PubMed, AJOG Glob Rep) - P3 | "In these exploratory analyses, wilate prophylaxis given 2-3 times a week without menstruation coordination was efficacious in reducing heavy menstrual bleeding in girls/women with VWD compared with on-demand treatment. Long-term prophylaxis has the potential to play a major role in improving the care and reducing the disease burden for girls/women with VWD."

Journal • Hematological Disorders • Hemophilia • Women's Health • IFNL1

Von Willebrand's late-life surprise in the ICU! (Euroanaesthesia 2025) - "A treatment with vWF concentrate was started in initial dose of 50 IU/kg, followed with 25 IU/kg every 12 hours until hemostasis was achieved along with intravenous tranexamic acid...It includes hemostatic therapies like desmopressin (DDAVP) or vWF concentrates (Wilate) for more severe cases...Blood product support, including red blood cell or platelet transfusions, is often required. It is necessary to collaborate with hematologists to optimize therapy and minimize complications.Learning points:1.VWD in the ICU presents significant challenge due to interaction with critical illnesses and coagulopathies.2.Early diagnosis, targeted therapy, and a multidisciplinary approach are important in reducing bleeding risks and improving outcomes.3.Balancing effective hemostasis with minimizing thromboembolic risks remains a key aspect of care for these vulnerable patients."

Cardiovascular • CNS Disorders • Hematological Disorders • Hemophilia

Plasma-Derived VWF/FVIII Concentrate (WILATE) Prophylaxis in Children and Adolescents With VWD (ASPHO 2025) - P3 | "pdVWF/FVIII prophylaxis was efficacious and well tolerated in children and adolescents with all types of VWD."

Clinical • Hemophilia

Effectiveness and Tolerability of Eqwilate in Real-life Conditions (clinicaltrials.gov) - P=N/A | N=38 | Active, not recruiting | Sponsor: Octapharma | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2021 ➔ Nov 2026 | Trial primary completion date: Sep 2021 ➔ May 2024

Enrollment closed • Trial completion date • Trial primary completion date

Efficacy and safety of plasma-derived von Willebrand factor/factor VIII concentrate (wilate) prophylaxis in children and adolescents with von Willebrand disease - WIL-31 study post hoc analysis. (PubMed, Res Pract Thromb Haemost) - "No serious adverse events related to study treatment or thrombotic events were observed. wilate prophylaxis was efficacious and well-tolerated in children and adolescents with all types of VWD."

Journal • Retrospective data • Hemophilia

Cost-effectiveness and cost-utility analysis of haemate-P versus other von willebrand disease treatments in Spain. (PubMed, J Med Econ) - "Treatment strategies compared included Haemate-P, Fanhdi, and Wilate in long-term prophylaxis (LTP) or on-demand treatment (ODT). Results were generally robust to sensitivity analyses. In patients with severe vWD experiencing a high bleed rate, Haemate-P prophylaxis is a less costly and potentially more effective treatment strategy and Haemate-P is cost-saving among on-demand strategies."

HEOR • Journal • Hematological Disorders • Hemophilia

Severe Thrombocytopenia in the Post-surgical Context and Administration of Factor VIIII (FVIII)/von Willebrand Factor (VWF) Concentrate in a Patient With von Willebrand Disease Type 2M. (PubMed, Cureus) - "Management of these patients may be challenging when trying to achieve the balance between avoiding the risk of haemorrhage and causing a risk of thrombosis with the treatment. We present a complex case of severe thrombocytopenia in a post-surgical setting, in which the timeline suggests a direct relationship between the worsening of platelet count and wilate administrations."

Journal • Cardiovascular • Hematological Disorders • Hemophilia • Thrombocytopenia • Thrombosis

Management of a hepatic laceration in a child with the Vicenza variant of Willebrand disease with Wilate® : Insights from an alsatian experience (EAHAD 2025) - No abstract available

Clinical

EMPOWER: A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients with VWD (clinicaltrials.gov) - P3 | N=20 | Recruiting | Sponsor: Unity Health Toronto | Not yet recruiting ➔ Recruiting

Enrollment open • Hemophilia

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

Efficacy of Wilate Prophylaxis in Reducing Nosebleeds in Patients with Severe VWD - A Post-hoc Analysis of the WIL-31 Study. (PubMed, Clin Appl Thromb Hemost) - "This post-hoc analysis demonstrated the efficacy of wilate prophylaxis in the prevention and treatment of nosebleeds in children and adults with severe VWD."

Journal • Retrospective data • Hemophilia

Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age (clinicaltrials.gov) - P3 | N=12 | Completed | Sponsor: Octapharma | Active, not recruiting ➔ Completed

Trial completion • Hemophilia

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

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

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

Leveraging National Real-World Evidence to Understand Differences of Economic Burden in Rare Diseases: The Case of Von Willebrand Disease in France (ISPOR-EU 2024) - "Within adult on-demand users, HRUC related to hospitalizations, in-hospital RT and FVIII, outpatient general practitioner and nurse visits, out-hospital RT and FVIII dispensings were assessed by AP and compared across RTs (Wilfactin ® , Veyvondi®, Voncento ® , Eqwilate ® ) using Generalized Estimating Equation models (GEE) accounting for intra-patient correlation and potential confounding factors. This large-scale real-world study is the first comparing costs related to the use of RT available in France. Costs were higher in AP treated with Wilfactin ® or Voncento ® than in those treated with Veyvondi ® ."

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

Von Willebrand Factor in Pregnancy (VIP) Study (clinicaltrials.gov) - P=N/A | N=110 | Recruiting | Sponsor: University of Washington | Trial completion date: Jun 2023 ➔ Dec 2026 | Trial primary completion date: Jun 2023 ➔ Dec 2026

Trial completion date • Trial primary completion date • Hemophilia

PREVAIL: Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq (clinicaltrials.gov) - P=N/A | N=14 | Terminated | Sponsor: Octapharma | N=80 ➔ 14

Enrollment change • Hematological Disorders • Hemophilia • Rare Diseases

Haemostatic management of von Willebrand disease during childbirth with a plasma-derived VWF/FVIII concentrate. (PubMed, J Thromb Haemost) - "The results of this case series indicate that wilate provided effective haemostatic cover for childbirth in females with VWD during delivery and postpartum."

Journal • Cardiovascular • Hemophilia • Postpartum Hemorrhage

Successful Management of Recurrent Ankle Hemarthrosis in a Patient with Von Willebrand Disease Type 3 using Emicizumab: A Case Report (ISTH 2024) - "Prophylactic treatments with Humate-P and Wilate had limited efficacy, leading to a high treatment burden and arthropathy. The patient's history involves a misdiagnosis of severe hemophilia A, intracranial hemorrhage, and the development of factor VIII inhibitors treated with immune tolerance therapy. Recurrent mucocutaneous bleeds, hemarthrosis, and a right ankle target joint significantly impacted the patient's quality of life. Genetic analysis confirmed a homozygous insertion mutation in the VWF gene."

Case report • Clinical • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Osteoarthritis • Rare Diseases

Bleeding Sites in von Willebrand Disease Patients on Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate – A Sub-Analysis of Data from the WIL-31 Study (ISTH 2024) - P3 | "Background: The WIL-31 study (NCT04052698) demonstrated the efficacy and safety of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (pdVWF/FVIII; wilate®) in people with von Willebrand disease (VWD)... Of 33 patients, six (18%) had severe type 1, 5 (15%) had type 2, and 22 (67%) had type 3 VWD. Under prophylaxis there were 173 breakthrough bleeds (62% spontaneous) resulting in a mean spontaneous ABR of 3.2 (86.9% reduction versus on-demand treatment). Adults and VWD type 1 patients had the highest number of total (6.3 and 9.0, respectively) and spontaneous (4.2 and 7.3, respectively) bleeds per patient across the age and type groups."

Clinical • Hemophilia

Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study (ISTH 2024) - P3 | "Background: The WIL-31 study (NCT04052698) demonstrated the efficacy and safety of regular prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (pdVWF/FVIII; wilate®) in people with von Willebrand disease (VWD)... Twenty-two type 3 VWD patients were included in the analysis population, with a median (range) age of 24 (7–61) years. The median weekly prophylactic dose was 62 IU/kg, administered twice weekly in 77% of patients. The mean total ABRs during prophylaxis were 3.9, 5.2 and 6.9 in children, adolescents and adults, respectively, vs 33.4, 32.0 and 40.0 during on-demand treatment, representing 88%, 84% and 86% reductions (Figure 1)."

Clinical • Hemophilia

EMPOWER: A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients With VWD (clinicaltrials.gov) - P3 | N=20 | Not yet recruiting | Sponsor: Unity Health Toronto | Initiation date: Jan 2024 ➔ Jun 2024

Trial initiation date • Hemophilia

Von Willebrand factor/factor VIII concentrate (wilate®) prophylaxis in children and adults with von Willebrand disease. (PubMed, Blood Adv) - P, P3 | "Overall, WIL-31 showed that wilate® prophylaxis was efficacious and well-tolerated in pediatric and adult patients with VWD of all types. The WIL-29 and WIL-31 trials were registered at www.clinicaltrials.gov as #NCT04053699 and #NCT04052698, respectively."

Journal • Hemophilia • Pediatrics • IFNL1

EMPOWER: A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients With VWD (clinicaltrials.gov) - P3 | N=20 | Not yet recruiting | Sponsor: Unity Health Toronto

New P3 trial • Hemophilia