FDA approves the first recombinant von Willebrand factor

FDA has approved recombinant von Willebrand factor (Vonvendi, Baxalta) for use in adult patients who have von Willebrand disease.

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Von Willebrand disease (VWD) is the most common inherited bleeding disorder, in which a person has a deficiency or impairment in the protein, von Willebrand factor an important component of the blood-clotting process. Patients with VWD can develop severe bleeding from the nose, gums and intestines, as well as into muscles and joints. It is estimated that approximately 1% of the US population has VWD, with men and women equally affected.

Vonvendi is the first FDA-approved recombinant von Willebrand factor, and is approved for as needed treatment and control of bleeding episodes in adults diagnosed with VWD. The treatment is also the first in the United States that contains only trace amounts of Factor VIII (FVIII), which allows for tailored treatment of patients who may not require additional FVIII.

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FDA's approval of Vonvendi was based on results from a phase 3 multicenter, clinical trial that assessed the safety and efficacy of Vonvendi with and without recombinant FVIII. In the study, 100% of participants reported successful treatment of bleeding episodes with 96.9% of treated bleeds achieving an “excellent” efficacy rating and the remainder achieving a “good” efficacy rating. Most bleeds were resolver with a single infusion and the treatment showed a mean half-life of 21.9 hours.

“With no major therapeutic innovation in more than a decade, Vonvendi offers patients an important new option for VWD with a clinical profile that can help them manage this challenging chronic disease,” says Joan Gill, MD, of the Blood Center and the Medical College of Wisconsin and lead author of the Vonvendi clinical study.

Generalized itching was most common adverse reaction observed and nsafety concerns were identified in the trials. 

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