Von Willebrand disease product approved in Europe

The European Commission has granted Shire marketing authorization for vonicog alfa (Veyvondi), a recombinant von Willebrand factor (rVWF) product.

The European Commission (EC) approved vonicog alfa for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults with von Willebrand disease (VWD) when desmopressin treatment alone is ineffective or not indicated.

The product was approved in the United States in 2015 for on-demand treatment and control of bleeding episodes in adults.

The approval means Shire is authorized to market vonicog alfa in the European Union as well as in Iceland, Lichtenstein, and Norway.

The EC’s approval of vonicog alfa was based on outcomes from three clinical trials. This includes a phase 1 study and a pair of phase 3 trials – one in a surgical setting and one in a nonsurgical setting.

Data from these studies are available in the Summary of Product Characteristics for vonicog alfa.The phase 1 trial (NCT00816660) enrolled patients with type 3 or severe type 1 VWD.

The goal was to assess the safety and pharmacokinetics of vonicog alfa combined at a fixed ratio with recombinant factor VIII – referred to as “rVWF-rFVIII.” The researchers compared rVWF-rFVIII with plasma-derived (pd) VWF combined with pdFVIII (pdVWF-pdFVIII).