Vonvendi (vonicog alfa) / Takeda - LARVOL DELTA

A Rare Combination of von Willebrand Disease Type 2A and 2M: Diagnostic and Therapeutic Challenges - A case report. (PubMed, Res Pract Thromb Haemost) - "Treatment with recombinant VWF (Veyvondi) restored high-molecular-weight multimers and achieved hemostatic control...This case demonstrates how AVWS may obscure an underlying congenital VWD phenotype. Combined phenotypic and molecular diagnostics are essential for accurate classification, therapeutic decision making, and interpretation of complex VWD phenotypes."

Journal • Cardiovascular • Gastroenterology • Hematological Disorders • Hemophilia

Spontaneous intradural extramedullary hematoma after mild exercise in Von Willebrand disease: A rare clinical presentation and literature review. (PubMed, Surg Neurol Int) - "Urgent surgical evacuation through T3/4 fenestration was performed, followed by targeted hemostatic therapy with recombinant von Willebrand factor (Vonicog Alfa)...SIEH should be considered in VWD patients presenting with acute spinal symptoms, even without trauma. Early MRI, prompt decompression, and tailored coagulation management are critical to optimal outcomes."

Journal • Back Pain • CNS Disorders • Hematological Disorders • Hemophilia • Mood Disorders • Musculoskeletal Pain • Pain

Targeting the VWF D4 Domain with a Humanized Monoclonal Antibody Partially Inhibits Shear-Induced Proteolysis Under Mechanical Circulatory Support (ISTH 2026) - "To overcome the lack of antibody cross-reactivity with porcine VWF, we utilized an in vivo heterologous minipig model supplemented with intravenous recombinant human VWF (rhVWF; Veyvondi®, Takeda; 100 IU/kg)...This represents a promising therapeutic strategy to mitigate acquired VWF HMW multimer defect and associated bleeding in patients requiring MCS. Table or Figure Upload (1) Page 2 Table or Figure Upload (2) DOI*10.1016/j.rpth.2026.104007"

Targeted Protein Degradation

Recombinant von Willebrand factor (rVWF) for surgical haemostasis and bleeding management: a 5-year retrospective review from a UK Haemophilia Centre (ISTH 2026) - "Background Recombinant von Willebrand factor (rVWF; vonicog alfa) has been approved in the UK since September 2020 for adults with von Willebrand disease (VWD) when desmopressin is ineffective or contraindicated...FVIII was used in 63% (5/8) and tranexamic acid in all cases...Demographics, perioperative haemostasis and bleeding management Table or Figure Upload (2) Figure 1. Median laboratory assay levels in pre- and post-infusion of rVWF with or without rFVIII DOI*10.1016/j.rpth.2026.106052"

Retrospective data • Review • Hematological Disorders • Hemophilia • Obstetrics • Rare Diseases • Thrombosis

Use of Recombinant von Willebrand Factor (Vonicog alfa) for Bleed Treatment in Children under 12 – a single centre experience (ISTH 2026) - "Bleeding episodes either due to trauma or surgery can be treated with a combination of tranexamic acid, desmopressin or von Willebrand factor (VWF) replacement...All children with a baseline FVIII:c <30iu/dL were given a single dose of recombinant FVIII (Octocog alfa) alongside the initial dose of Vonicog alfa...Table or Figure Upload (1) Patient demographics and baseline von Willebrand parameters Page 2 Table or Figure Upload (2) Indication, dosing and outcomes for patients treated with Vonicog alfa. Page 3 DOI*10.1016/j.rpth.2026.105059"

Clinical • Hematological Disorders • Hemophilia • Immunology

Use of Recombinant von Willebrand Factor (Vonicog Alfa) for Prophylaxis in a Child with Type 3 von Willebrand Disease (ISTH 2026) - "Graph produced by Copilot-AI, reviewed for accuracy. Page 2 DOI*10.1016/j.rpth.2026.105086"

Clinical • Hematological Disorders • Hemophilia • Immunology • Mood Disorders

Elongational Strain Rate Drives VWF Activation and Thrombosis via the A1-GPIbα Axis in Complex Geometries Independent of Wall Shear Rate (ISTH 2026) - "Methods We perfused fluorescent rVWF (VONVENDIÒ) or whole blood over type I collagen through microfluidic cross-slots, bifurcations, and stenosis models...(c) Thrombi in low- WSR/high-ESR regions; formation is abrogated by AK2. DOI*10.1016/j.rpth.2026.103500"

Cardiovascular • Hematological Disorders • Thrombosis

Detection of Anti-VWF Antibodies in Patients with Acquired von Willebrand Disease: Case Studies (ISTH 2026) - "96-well plates were coated with either rVWF (VONVENDI®) or carbonate buffer alone...Table or Figure Upload (1) Table 1. Case Results Summary Page 2 DOI*10.1016/j.rpth.2026.104016"

Case study • Clinical • Cardiovascular • Endocrine Disorders • Hematological Malignancies • Hemophilia • Immunology

A Comprehensive Disproportionality Analysis of Drug-Related Head Injury Reports Using the FAERS Database. (PubMed, Brain Behav) - "This large-scale pharmacovigilance study identifies a strong association between coagulation factor therapies and reports of head injury. This signal most plausibly reflects confounding by indication rather than a direct prothrombotic mechanism. It also raises safety concerns regarding chamomile. These findings highlight the need for increased clinical vigilance and further investigation into these potential risks."

Journal • Retrospective data

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=47 | Completed | Sponsor: Baxalta now part of Shire | Recruiting ➔ Completed | N=31 ➔ 47

Enrollment change • Trial completion • Hemophilia

Real-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study. (PubMed, J Comp Eff Res) - " Among 2540 on-demand RT users, WILFACTIN® was the main RT used, followed by VONCENTO®, VEYVONDI®, EQWILATE® and WILSTART®. This first real-world study suggests that VEYVONDI® seems to be a cost-saving RT compared with other RT. Future studies including clinical data should provide further evidence."

Journal • Hemophilia

Type 2N von Willebrand Disease in Pregnancy and Postpartum: A Case Report (THSNA 2026) - "Due to the high-risk nature of her pregnancy and poor outcome from the previous 2 pregnancies, treatment was initiated with recombinant vWF replacement (VonvendiÒ) biweekly to maintain hemostasis antepartum. Frequent infusions and inadequate FVIII recovery led to treatment transition to efanesoctocog alfa (AltuviiioÒ) at 17 weeks gestation with goal FVIII trough of >10%...Re-bleeding occurred after discontinuation of treatment and required additional therapy about every 2-3 weeks with WilateÒ and tranexamic acid...Further investigation and research should be conducted on the bleeding tendencies of patients with 2N von Willebrand Disease, especially surrounding pregnancy and postpartum care. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Case report • Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Comparative Clinical and Economic Evaluation of On-Demand versus Prophylactic Treatment with pdVWF/FVIII 1:1 and Recombinant VWF in Adults with Type 3 von Willebrand Disease in the United States (THSNA 2026) - P3 | "In the United States, plasma-derived VWF/FVIII in a 1:1 activity ratio (pdVWF/FVIII 1:1; wilate®) and recombinant VWF (rVWF; Vonvendi®) are the only concentrates approved for prophylaxis. wilate prophylaxis provides robust clinical benefits and significant cost advantages compared with OD therapy and rVWF prophylaxis in adults with severe type 3 VWD. These findings, supported by prospective intrapatient data and current U.S. pricing, indicate that wilate is a cost-efficient, first-line prophylactic option from both payer and societal perspectives in the US. Future analyses incorporating quality-adjusted life years and indirect health outcomes (e.g., joint preservation, chronic anemia prevention) will further define the long-term economic and therapeutic value of prophylaxis in this population."

Clinical • HEOR • Hematological Disorders • Hemophilia • IFNL1

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Recruiting | Sponsor: Baxalta now part of Shire | Trial primary completion date: Jul 2019 ➔ May 2020 | Trial completion date: Jul 2019 ➔ May 2020

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Recruiting | Sponsor: Baxalta now part of Shire | Not yet recruiting ➔ Recruiting | Initiation date: Nov 2016 ➔ Dec 2017

Enrollment open • Trial initiation date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Not yet recruiting | Sponsor: Baxalta US Inc.

New P3 trial • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=31 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Jan 2025 ➔ Mar 2026 | Trial primary completion date: Jan 2025 ➔ Mar 2026

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=34 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Mar 2024 ➔ Jan 2025 | Trial primary completion date: Mar 2024 ➔ Jan 2025

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=34 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Mar 2023 ➔ Mar 2024 | Trial primary completion date: Mar 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=34 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Dec 2022 ➔ Mar 2023 | Trial primary completion date: Jun 2022 ➔ Mar 2023

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=34 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Dec 2021 ➔ Dec 2022 | Trial primary completion date: Dec 2021 ➔ Jun 2022

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Recruiting | Sponsor: Baxalta now part of Shire | Active, not recruiting ➔ Recruiting

Enrollment open • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Active, not recruiting | Sponsor: Baxalta now part of Shire | N=24 ➔ 39 | Trial completion date: Jun 2020 ➔ Dec 2021 | Trial primary completion date: Jun 2020 ➔ Dec 2021

Enrollment change • Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=24 | Active, not recruiting | Sponsor: Baxalta now part of Shire | Recruiting ➔ Active, not recruiting

Enrollment closed • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=24 | Recruiting | Sponsor: Baxalta now part of Shire | N=39 ➔ 24 | Trial completion date: Nov 2020 ➔ Jun 2020 | Trial primary completion date: Nov 2020 ➔ Jun 2020

Enrollment change • Trial completion date • Trial primary completion date • Hemophilia