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Vonvendi, the first recombinant treatment for adults with von Willebrand disease

The FDA ( Food and Drug Administration ) has approved Vonvendi ( von Willebrand factor [ recombinant ] ). Vonvendi is the first and only recombinant treatment for adults living with von Willebrand Disease ( VWD ).

Vonvendi is an innovative recombinant protein treatment that includes a physiologic distribution of proteins called multimers, including ultra-large multimers ( ULMs ), with large multimers being the most active form of the protein supporting clot formation.
The treatment is also the first in the United States that contains only trace amounts of Factor VIII ( FVIII ), offering the flexibility to administer FVIII only when needed. This attribute allows for tailored treatment for patients who may not require additional FVIII.

VWD is the most common inherited bleeding disorder worldwide, affecting up to one in 100 people; the rarest and often most severe form affects one in 1,000,000 people worldwide. This genetic disorder causes alterations or deficits in von Willebrand factor ( VWF ), resulting in impaired clotting, and affects women and men equally.
The disease can manifest through a variety of bleeding events, including mucosal bleeds, gastrointestinal bleeds or menorrhagia. Patients often live with the disease for years without a proper diagnosis; even with a confirmed diagnosis, there are limited treatment options available.

Vonvendi has been approved for on-demand treatment and control of bleeding episodes in adults with von Willebrand disease.
The FDA approval was based on positive results from a phase III multicenter, open-label clinical trial that assessed the safety, efficacy and pharmacokinetics of Vonvendi with and without recombinant FVIII.
In the pivotal study, all participants ( 100% ) reported successful treatment of bleeding episodes, with 96.9% of treated bleeds ( n=192 bleeds in 22 patients ) achieving an excellent efficacy rating and 3.1% achieving a good efficacy rating.
Most bleeds ( 81.8% ) were resolved with a single infusion, and the treatment showed a mean half-life of 21.9 hours ( ± 8.36 ).

No thrombotic events or severe product-related adverse events were observed during the clinical trial, nor were there treatment-related binding or neutralizing antibodies against VWF or neutralizing antibodies against FVIII.
The most common adverse reaction observed in greater than two percent of patients in clinical trials was generalized pruritus. ( Xagena )

Source: Baxalta, 2015