VONVENDI Approved to Treat Von Willebrand Disease

The U.S. Food and Drug Administration has approved Shire’s VONVENDI, a recombinant von Willebrand factor treatment for perioperative management of bleeding in adults 18 years and older with von Willebrand disease (VWD). VONVENDI is also indicated for on demand treatment and control of bleeding episodes, and it is the first and only recombinant treatment for adults living with VWD, the most common inherited bleeding disorder.

Approval is based on results from a Phase III prospective, open-label, multicenter trial that evaluated the efficacy and safety of VONVENDI with or without recombinant factor VIII treatment in elective surgical procedures in adults 18 years and older diagnosed with severe VWD. Results showed VONVENDI met its primary endpoint demonstrating overall hemostatic efficacy assessed 24 hours after the last perioperative VONVENDI infusion or at completion of study visit, whichever occurred earlier. The overall median dosing frequency of once-daily was demonstrated to normalize hemostasis in appropriate patients. One study participant developed deep vein thrombosis three days after undergoing hip replacement surgery while receiving VONVENDI.

In addition to the expanded use of VONVENDI, the updated prescribing information states the product can be stored at refrigerated temperature 2 degrees Celsius (36 degrees Fahrenheit to 46 degrees Fahrenheit) or room temperature not to exceed 30 degrees Celsius (86 degrees Fahrenheit).

“Persons with von Willebrand disease face a heightened risk of bleeding during surgery and may require factor treatment before, during or after surgery,” said Michael Tarantino, MD, professor of pediatrics and medicine at the University of Illinois College of Medicine and medical director and president of the Bleeding and Clotting Disorders Institute. “For surgeries requiring repeated, frequent infusions with combined von Willebrand factor and factor VIII concentrates, an excessive rise in factor VIII levels may increase the risk of thromboembolic complications such as blood clots. The expanded use of VONVENDI in surgical settings gives healthcare professionals flexibility in treating von Willebrand disease with an appropriate dose of von Willebrand factor, with or without recombinant factor VIII, based on each patient’s unique needs.”