Back to discover
Disease

Von Willebrand disease

Late-stage therapeutic development

Also known as Hereditary von Willebrand disease.

19
Clinical trials
6
Associated genes
3
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 4 clinical trials expected to report results, the earliest in Q2 2027.
Upcoming Milestones
Clinical Milestones6View
Results posted 1
Results expected 4
Recently completed 1
Activity timeline6

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Vonicog alfaapproved

Original application approval — Prevention and treatment of haemorrhage or surgical bleeding in adults (age 18 years and… (2018)

Clinical trials

15 sponsors · 0 new · 1 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
11
Late-stage
6
Completed
Late-stage studies

A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease

Phase 3 · Recruiting · Takeda

A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients With Type 3 Von Willebrand Disease

Phase 3 · Recruiting · Hoffmann-La Roche

A Post-marketing Observational Study to Assess the Efficacy and Safety of the FVIII/VWF Complex (Human), Alphanate®, in Preventing Excessive Bleeding During Surgery in Subjects With Congenital Type 3 Von Willebrand Disease

Phase 4 · Active, not recruiting · Grifols Biologicals, LLC

Severe Aortic Stenosis and Acquired Von Willebrand´s Disease: The Impact of Desmopressin in Valve-Replacement Surgery

Phase 4 · Completed · Pontificia Universidad Catolica de Chile

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease

Phase 3 · Completed · Baxalta now part of Shire

A Phase 3b, Prospective, Open-Label, Uncontrolled, Multicenter Study on Long-Term Safety and Efficacy of rVWF in Pediatric and Adult Subjects With Severe Von Willebrand Disease (VWD)

Phase 3 · Completed · Baxalta now part of Shire
Recruiting

A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease

Phase 3 · Recruiting · Takeda

A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients With Type 3 Von Willebrand Disease

Phase 3 · Recruiting · Hoffmann-La Roche

Emicizumab for Severe VON Willebrand Disease (VWD) and VWD/Hemophilia A

Phase 1 · Recruiting · Bleeding and Clotting Disorders Institute Peoria, Illinois

Interest of Sublingual Videomicroscopy for the Prediction of Bleeding in Von Willebrand Disease and Other Constitutional Haemorrhagic Diseases

N/A · Recruiting · University Hospital, Lille
Recently completed

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease

Phase 3 · Completed · Baxalta now part of Shire

A Phase 3b, Prospective, Open-Label, Uncontrolled, Multicenter Study on Long-Term Safety and Efficacy of rVWF in Pediatric and Adult Subjects With Severe Von Willebrand Disease (VWD)

Phase 3 · Completed · Baxalta now part of Shire

Clinical Study to Investigate the Efficacy, Pharmacokinetics, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease Patients Under the Age of 6 Years

Phase 3 · Completed · Octapharma

Prospective, Randomized Trial Comparing Recombinant Von Willebrand Factor (rVWF) Plus Tranexamic Acid vs. rVWF Alone to Reduce Postpartum Hemorrhage in Women With Von Willebrand Disease: The VWD-WOMAN Trial

Phase 3 · Completed · Nicoletta C Machin

Phase II Biologic Effects Study of Recombinant Interleukin-11 (rhIL-11, Neumega) in Subjects With Moderate or Mild Hemophilia A, or Von Willebrand Disease Unable to Use DDAVP

Phase 2 · Completed · University of Pittsburgh

Severe Aortic Stenosis and Acquired Von Willebrand´s Disease: The Impact of Desmopressin in Valve-Replacement Surgery

Phase 4 · Completed · Pontificia Universidad Catolica de Chile

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

Product approvals and authorisations
2018emaOriginal application approval — Vonicog alfa· Prevention and treatment of haemorrhage or surgical bleeding in adults (age 18 years and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or contraindicated. Veyvondi should not be used in the treatment of Haemophilia A. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

6 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

VWF0.83DOCK90.26NTF30.21PTMS0.13CARTPT0.13TREML10.05

Disease biology

3 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

von Willebrand factorvia VWF0.83Neurotrophin-3via NTF30.21Cocaine- and amphetamine-regulated transcript proteinvia CARTPT0.13

Reference

Authoritative identity, definition & identifiers.

Synonyms

Hereditary von Willebrand disease

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.