European Commission approves Veyvondi to treat von Willebrand disease.- Shire.

Shire plc announced that the European Commission (EC) has granted Marketing Authorization for Veyvondi [vonicog alfa, recombinant von Willebrand factor] (rVWF), for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin (DDAVP) treatment alone is ineffective or not indicated. Veyvondi should not be used in the treatment of hemophilia A.

Veyvondi is the first and only recombinant von Willebrand Factor (rVWF) treatment in the EU for von Willebrand disease (VWD) that specifically addresses the primary deficiency or dysfunction of von Willebrand Factor (VWF) while also allowing the body to restore and maintain adequate Factor VIII (FVIII) plasma levels. The Marketing Authorization is based on outcomes from three clinical trials of a total 80 patients with VWD exposed to Veyvondi. These include a Phase 1 multicenter, controlled, randomized, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics (PK) of rVWF:rFVIII in subjects 18 to 60 years of age with severe VWD; a Phase III multicenter, open-label study to assess the PK, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in adult subjects with severe VWD; and a Phase III, prospective, open-label, uncontrolled, non-randomized, international multicenter study to assess the hemostatic efficacy and safety of rVWF with or without rFVIII in 15 adult subjects with severe VWD undergoing major, minor, or oral elective surgical procedures.

VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population or approximately 100,000 people in the EU. VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting. Only a minor proportion of affected individuals have the severe form of the disease and are in need of VWF replacement.6 Symptoms range from nosebleeds to bleeding from the gums and easy bruising. Bleeding from the stomach and intestines can occur but is less common.

Comment: In the US the product has been approved for more than two years under the trade name Vonvendi [von Willebrand factor (Recombinant)] and is indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease for on-demand treatment and control of bleeding .