Recombinant von Willebrand factor: a first-of-its-kind product for von Willebrand disease

Department

Internal Medicine

Document Type

Article

Publication Title

Drugs of Today (Barcelona, Spain : 1998)

Abstract

von Willebrand disease (VWD) is caused by quantitative or qualitative defects in von Willebrand factor (VWF). The mainstay of therapy is desmopressin, which is, however, not useful in certain forms of VWD notwithstanding adverse events. For these patients, plasma-derived factor VIII (pdFVIII)/VWF concentrates have been available for close to three decades but have a theoretical risk of disease transmission, hypersensitivity/allergic reactions, inhibitors and thrombosis. A recombinant VWF (vonicog alfa, Vonvendi™; manufactured by Baxalta, now part of Shire) was approved by the U.S. Food and Drug Administration (FDA) in December 2015. This review will survey the literature based on a MEDLINE review on the safety, efficacy and pharmacokinetics of Vonvendi. It will also summarize the ongoing studies on Vonvendi available in the public domain. Vonvendi may have an important role in the management of VWD. However, more studies are needed, especially in special populations such as surgical patients, patients with major gastrointestinal bleeding from arteriovenous malformations and pregnant women and children, who are most likely to benefit from it.

First Page

653

Last Page

664

DOI

10.1358/dot.2016.52.12.2570978

Volume

52

Issue

12

Publication Date

12-1-2016

Medical Subject Headings

Child; Deamino Arginine Vasopressin (therapeutic use); Female; Humans; Pregnancy; Recombinant Proteins (adverse effects, pharmacokinetics, therapeutic use); von Willebrand Diseases (drug therapy); von Willebrand Factor (adverse effects, pharmacokinetics, therapeutic use)

PubMed ID

28276537

Link to Full Text

Share

COinS