Press release
Von Willebrand Disease Treatment Market Size in the 7MM is expected to grow by 2034, estimates DelveInsight
Von Willebrand Disease Market
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Key Takeaways from the Von Willebrand Disease Market Report
• In March 2025, Takeda conducted a study is to evaluate the effectiveness of prophylaxis with vonicog alfa (recombinant von Willebrand factor [rVWF]) in children. This study will enroll those participants who have been previously treated with VWF product or with a plasma-derived VWF (pdVWF) product. In this study, participants will be treated with vonicog alfa (rVWF) for 12 months.
• In March 2025, Hemab ApS announced a phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants With Von Willebrand Disease (Velora Pioneer). This is a first-in-human (FIH), Phase 1/2, open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study of HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) design to establish safety, tolerability, PK, and PD effect. In Part B of the study, the safety and tolerability of repeat dosing will be established prior to cohort expansion to explore efficacy.
• In 2023, the total Von Willebrand Disease diagnosed prevalent cases in the US were approximately 13,000, cases, which are expected to increase during the study period (2020-2034).
• Females predominantly account for around 55% of Von Willebrand Disease diagnosed prevalent cases in the US, making up more than 7,000 cases in 2023.
• Age group 5-13 years and 14-18 years accounts for around 32% and 22% of all Von Willebrand Disease diagnosed prevalent cases in the US, in 2023. Whereas, age groups 0-4, 19-44, and >45 years reported around 6%, 23%, and 17% cases, respectively, in 2023.
• Von Willebrand disease Type 2 and Type 3 accounts for around 11% and 6% of all Von Willebrand Disease diagnosed prevalent cases in the US, in 2023. Whereas, von willebrand disease Type 1 reported around 83% cases alone, in 2023.
• The leading Von Willebrand Disease Companies such as Octapharma, Grifols Therapeutics LLC, CSL Behring, Margaret Ragni, Shire, Archemix Corp., and others.
• Promising Von Willebrand Disease Therapies such as Wilate, plasma-derived FVIII/VWF concentrate, Biostate®, Voncento, Oprelvekin, Interleukin 11, IL-11, Alphanate SD/HT, rVWF, ARC1779, and others.
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Von Willebrand Disease Epidemiology Segmentation in the 7MM
• Total Von Willebrand Disease diagnosed prevalent cases
• Von Willebrand Disease Gender-specific diagnosed prevalent cases
• Von Willebrand Disease Type-specific diagnosed prevalent cases
• Von Willebrand Disease Age-specific diagnosed prevalent cases
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Von Willebrand Disease Marketed Drugs
• WILATE: OctaPharma
WILATE is the first von Willebrand factor (VWF) concentrate indicated for prophylactic treatment across all forms of Von Willebrand disease, marking a significant milestone in the field. In December 2023, Octapharma USA announced the expansion of the US FDA approval for WILATE, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection. The approved label now includes routine prophylaxis aimed at reducing the frequency of bleeding episodes in adults and children aged 6 and older with any type of von Willebrand disease, the most prevalent bleeding disorder in the United States. The FDA approval is supported by Octapharma's WIL-31 study, a prospective, non-controlled, international, multicenter Phase III trial that investigated the efficacy and safety of wilate prophylaxis over 12 months in people aged 6 and older with severe Von Willebrand disease of any type.
• VONVENDI: Takeda Pharmaceuticals
VONVENDI is a recombinant von Willebrand factor replacement therapy. It is approved for the treatment to reduce the frequency of bleeding episodes for severe type 3 von Willebrand disease. In January 2022, US FDA approved VONVENDI [von Willebrand Factor (Recombinant)] for routine prophylaxis treatment in patients with severe Type 3 von Willebrand disease receiving on-demand therapy. The approval was based on data from a prospective, open-label, international multicenter study that evaluated efficacy and safety of prophylactic treatment. Investigators observed whether frequency of bleeding episodes were reduced in 10 adult patients diagnosed with severe Type 3 von Willebrand disease, who were previously treated on-demand.
Von Willebrand Disease Emerging Drugs
• VGA039: Vega Therapeutics
Vega Therapeutics' VGA039 is a first-in-class antibody therapy with a novel mechanism of action that modulates Protein S - a key co-factor involved in thrombin generation during both initiation and propagation of coagulation - with preclinical data demonstrating efficacy in numerous congenital bleeding disorders, including von Willebrand disease. By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in Von Willebrand disease and, as a subcutaneously self-administered antibody therapy, has potential to transform von Willebrand disease treatment. It is currently recruiting in a Phase I study to assess safety, tolerability, pharmacokinetic, and pharmacodynamics study of VGA039 following IV or SC administration of single ascending doses in healthy adults and subcutaneous adult patients with von willebrand disease. In May 2023, the US FDA granted VGA039 Orphan Drug Designation.
• Efanesoctocog alfa (BIVV001): Sanofi
Efanesoctocog alfa (BIVV001), is a novel and investigational recombinant factor VIII therapy. It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to extend its time in circulation. It is the first investigational factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It is being developed by Sanofi to assess the pharmacokinetics and safety and tolerability of efanesoctocog alfa (BIVV001) in adults with Type 2N and 3 Von Willebrand disease. It is currently under Phase I development.
To learn more about Von Willebrand Disease Treatment guidelines, visit @ Von Willebrand Disease Treatment Market Landscape- https://www.delveinsight.com/sample-request/von-willebrand-disease-vwd-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Von Willebrand Disease Market Outlook
The current Von Willebrand Disease therapeutics market landscape in the United States can be divided into three major categories based on the class of drugs administered, they are; Non-replacement Therapy using desmopressin (DDAVP) to increase the plasma concentration of vWF, Replacement Therapy using plasma-derived coagulation factor concentrates (HUMATE-P, ALPHANATE, IMMUNATE, and WILATE) and Adjunct Therapy using primarily anti-fibrinolytics, such as Tranexamic Acid (CYCLOKAPRON).
Von Willebrand Disease Therapies and Companies
• Wilate: Octapharma
• plasma-derived FVIII/VWF concentrate: Grifols Therapeutics LLC
• Biostate®: CSL Behring
• Voncento: CSL Behring
• Oprelvekin, Interleukin 11, IL-11: Margaret Ragni
• Alphanate SD/HT: Grifols Biologicals, LLC
• rVWF: Baxalta now part of Shire
• ARC1779: Archemix Corp.
Learn more about the FDA-approved drugs for Von Willebrand Disease @ Drugs for Von Willebrand Disease Treatment- https://www.delveinsight.com/sample-request/von-willebrand-disease-vwd-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Scope of the Von Willebrand Disease Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Von Willebrand Disease Companies- Octapharma, Grifols Therapeutics LLC, CSL Behring, Margaret Ragni, Shire, Archemix Corp., and others.
• Von Willebrand Disease Therapies- Wilate, plasma-derived FVIII/VWF concentrate, Biostate®, Voncento, Oprelvekin, Interleukin 11, IL-11, Alphanate SD/HT, rVWF, ARC1779, and others.
• Von Willebrand Disease Therapeutic Assessment: Von Willebrand Disease Current marketed and Von Willebrand Disease emerging therapies
• Von Willebrand Disease Market Dynamics: Von Willebrand Disease market drivers and Von Willebrand Disease market barriers
• Von Willebrand Disease Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Von Willebrand Disease Unmet Needs, KOL's views, Analyst's views, Von Willebrand Disease Market Access and Reimbursement
Discover more about Von Willebrand Disease Drugs in development @ Von Willebrand Disease Clinical Trials Assessment- https://www.delveinsight.com/sample-request/von-willebrand-disease-vwd-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Table of Content
1. Von Willebrand Disease Market Report Introduction
2. Executive Summary for Von Willebrand Disease
3. SWOT analysis of Von Willebrand Disease
4. Von Willebrand Disease Patient Share (%) Overview at a Glance
5. Von Willebrand Disease Market Overview at a Glance
6. Von Willebrand Disease Disease Background and Overview
7. Von Willebrand Disease Epidemiology and Patient Population
8. Country-Specific Patient Population of Von Willebrand Disease
9. Von Willebrand Disease Current Treatment and Medical Practices
10. Von Willebrand Disease Unmet Needs
11. Von Willebrand Disease Emerging Therapies
12. Von Willebrand Disease Market Outlook
13. Country-Wise Von Willebrand Disease Market Analysis (2020-2034)
14. Von Willebrand Disease Market Access and Reimbursement of Therapies
15. Von Willebrand Disease Market Drivers
16. Von Willebrand Disease Market Barriers
17. Von Willebrand Disease Appendix
18. Von Willebrand Disease Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
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