ASHLEY FLETCHER
Ashley Fletcher is the AHCDO Australian Bleeding Disorders Registry (ABDR) Senior Research Fellow.
Von Willebrand disease (VWD) is a lifelong bleeding disorder caused by problems with a blood protein called von Willebrand factor (VWF), which helps blood clot properly. Current treatments are effective for many people, but researchers around the world are working on new therapies that may offer more options in the future, especially for people with more severe or complex forms of VWD.
In recent articles I have concentrated on novel and emerging therapies in haemophilia, with less on VWD. But at the Blood 2025 conference there was a good session on VWD and particularly the presentation by Prof Frank Leebeek from Erasmus University Medical Centre in the Netherlands on Treatment options for von Willebrand disease in the near future.
Below is an overview of novel and emerging treatments currently being studied. Most of these are experimental and not yet approved for routine use, but they show promising directions for future care.
NB: ‘Current stage’ relates to the completed clinical trials presented at the Blood 2025 Conference.
| Efanesoctocog alfa (BIVV-001) |
| How it’s given: Intravenous (into a vein). |
| How it works: Increases levels of clotting factor VIII (8), which works closely with VWF. |
| Current stage: Early clinical trials (Phase 1) in people with VWD type 2N and type 31. |
| What this means for patients: This treatment may help people with very low factor VIII levels, but results are still awaited. |
| Emicizumab2 |
| How it’s given: Subcutaneous injection (under the skin). |
| How it works: Acts like factor VIII using a laboratory-made antibody. |
| Current stage: Approved for haemophilia A, but not approved for VWD. |
| What this means for patients: Some doctors have used it ‘off label’ (prescribed but not yet officially approved for that condition) in selected VWD cases, but it is not yet a standard VWD treatment. |
| VGA0393 |
| How it’s given: Subcutaneous injection. |
| How it works: A monoclonal antibody that affects a natural blood protein (protein S) to improve clotting. |
| Current stage: Phase 1 studies in healthy volunteers and people with VWD. |
| What this means for patients: Very early research but shows potential as a new way to reduce bleeding. |
| Rondaptivo pegol (BT200)4 |
| How it’s given: Subcutaneous injection. |
| How it works: Binds to part of the VWF protein to improve how it works. |
| Current stage: Clinical studies in people with VWD types 2A and 2B |
| What this means for patients: One of the more advanced experimental treatments specifically designed for certain VWD subtypes. |
| Nanobody KB-V13A125 |
| How it’s given: Subcutaneous injection. |
| How it works: Links VWF to another blood protein (albumin) to keep it working longer. |
| Current stage: Animal studies (mouse models). |
| What this means for patients: Promising science, but still far from human use. |
| Synthetic Nanoparticles |
| How they’re given: Intravenous. |
| How they work: Help VWF bind to blood vessels and platelets. |
| Current stage: Animal studies only. |
| What this means for patients: Early research exploring completely new treatment concepts. |
| siRNA (Gene Silencing Therapy)6 |
| How it’s given: Subcutaneous injection. |
| How it works: Turns off problem genes that cause abnormal VWF |
| Current stage: Preclinical studies in mice with VWD type 2B |
| What this means for patients: A potential way to target the genetic cause of disease but not yet tested in people. |
| Gene Editing (CRISPR/Cas9) |
| How it works: Directly edits DNA to correct or switch off problem VWF genes. |
| Current stage: Animal studies (dogs) and laboratory cell studies. |
| What this means for patients: A possible long-term or permanent treatment in the distant future, but still highly experimental. |
If you are interested in new or experimental treatments, talk to your Haemophilia Treatment Centre or haematology specialist. They can explain what is currently available and whether any of the clinical trials may be suitable for you.
Research continues to bring hope for more personalised and effective treatments for people living with von Willebrand disease.
To find out more, there is an excellent overview of current and emerging therapies as potential treatment for people with von Willebrand disease, which one of our HTC Directors, Assoc Prof Jennifer Curnow, co-authored with her Westmead Hospital colleagues pathologist Prof Emmanuel Favaloro and haematologist Dr Leonardo Pasalic. If you would like to read the full article and don’t have access via a university library, speak to your state library about their interlibrary loan service, where they provide single journal articles for a fee.
3.Millar CM, Raheja P, Wheeler AP, et al. A Phase Ia study of VGA039, a protein s–targeting monoclonal antibody, in individuals with von Willebrand disease demonstrates concentration-dependent increases in thrombin generation and potential for subcutaneous prophylactic dosing. In: 66th American Society of Hematology Annual Meeting and Exposition. Orlando, FL: American Society of Hematology; 2024.
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