Von Willebrand disease – increasing awareness and access to vital care
pharmafile | November 4, 2025 | Feature | Medical Communications, Research and Development | European Medicines Association, Haematology, Takeda, Von Willebrand Disease, clinical trials, disease awareness
Pharmafile talks to Anthea Cherednichenko, Vice President Franchise Head Haematology and Transplant at Takeda about Von Willebrand disease, its causes and symptoms, treatments in development and why raising greater awareness of the condition is so important.
Pharmafile: Can you give us an overview of Von Willebrand disease (VWD) and its symptoms?
Anthea Cherednichenko (AC): VWD is one of the most prevalent bleeding disorders, impacting around 1% of the population –about three million people in the US – and it affects men and women approximately equally. People with VWD either don’t have or don’t produce enough Von Willebrand factor (VWF) – one of many factors that enables blood to clot within the body – meaning that blood doesn’t clot within the normal time frame. VWD is an inherited disease and symptoms can range from severe nose bleeds to severe and debilitating menstrual bleeding and life-threatening bleeds in the gastrointestinal area. For women, heavy periods are common and childbirth can be risky. Additionally, patients have a heightened risk of spontaneous bleeding events, including internally, and injuries can trigger bleeds that patients might not even notice immediately.
Pharmafile: What are the current treatments for VWD?
AC: Initially patients use a medication called desmopressin, which is designed to stimulate blood clotting. However, many patients need a more targeted therapy, one that works more effectively for severe bleeds. This involves treatment that introduces factor concentrate, which tries to replace the missing VWF. There are two types available currently: one is a plasma-derived factor concentrate, originating from human plasma; and the other is a recombinant Von Willebrand factor, called VONVENDI. VONVENDI can be used in adults and children with VWD to treat and control bleeding episodes (on-demand), to prevent excessive bleeding during and after surgery (perioperative) and for adult patients only, to reduce the number of bleeding episodes when used regularly (prophylaxis).
Pharmafile: What is limiting these current treatments and how can these obstacles be overcome?
AC: Unfortunately many patients go undiagnosed for many years, experiencing what we call a ‘diagnostic odyssey’ before receiving the correct diagnosis, which is the first challenge that needs to be addressed.
Even after patients have been diagnosed, problems persist. Patients can feel that their symptoms are being minimised – especially in the case of heavy menstrual bleeding, where some patients may be given hormone therapy, while others can be told that it is not serious enough to warrant a more involved therapy. Yet many of the symptoms are debilitating and can even be life threatening. Patients miss a lot of school or work or can’t contribute to society in a normal way, and we’ve heard some really tough stories about young women who miss a lot of school days because of heavy menstrual bleeding. So the next phase of treatment involves helping patients advocate for themselves and also to increase education about these symptoms.
One of the challenges is that the way patients experience these bleeds can be very diverse as, aside from the three main categories of nose bleeds, heavy menstrual bleeding and gastrointestinal bleeding, there are a number of ways that patients can be impacted by spontaneous or traumatic bleeds. Patients tend to feel that they can’t go out and live their lives due to their fear of having a bleed. This includes basic things such as attending school, pursuing their chosen career and being productive in the workplace, through to social interactions and being involved in their communities, particularly in terms of playing sport. The disease can put significant limits on patients because they feel forced to limit their activities due to the fear of having a bleed.
Another issue is the importance of ensuring that patients have equal access to care, which remains a challenge across many areas, and particularly in the case of rare diseases – in this patient population in particular, it’s difficult to create a pool of evidence to demonstrate the pressing need for these treatments.
Pharmafile: How does Vonvendi differs from other forms of therapy?
AC: Vonvendi launched in the US about ten years ago and remains the first and only recombinant VWF available. Other similar available treatments are plasma-derived, and therefore might contain some other factors as well. Vonvendi just replaces what is missing – the VWF – and it is the only recombinant product on the market for this treatment indication. Vonvendi is also unique in that it is effective over a 22-hour half-life, and in the studies the majority of patients treated on demand needed just one Vonvendi infusion to treat the bleed.
Pharmafile: Does Takeda have any other research pipelines looking at new therapies for VWD?
AC: Vonvendi – also known as Veyvondi’ in Europe – has ongoing applications to both the European Medicines Association (EMA) and Japan Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of children. More broadly, Takeda put forward some promising data at the International Society on Thrombosis and Haemostasis in July this year regarding one of our haemophilia A products, Adynovate, for use in previously untreated patients. We’re making progress in other bleeding disorders too, with a pipeline targeting the unmet need in the bleeding disorder space.
Pharmafile: How do you see treatment for VWD developing over the next five years?
AC: One of the most important things is to continue to raise awareness, to help improve patients’ diagnostic journeys. There is so much potential for patients to receive an optimal standard of care and I think that more patients, particularly children, will continue to be able to receive preventative treatment. There is a lot of opportunity for the standard of care to be elevated, with VBF and similar treatments, in order to give these patients a more promising future.
Anthea Cherednichenko is Vice President Franchise Head Hematology and Transplant at Takeda. The Japanese biopharma develops therapeutics in oncology, rare diseases, neuroscience, gastrointestinal and inflammation.
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