Cambridge, Ma. -- Shire plc, the global biotechnology leader in rare disease, today announced the U.S. Food and Drug Administration (FDA) has approved VONVENDI [von Willebrand factor (recombinant)], a recombinant von Willebrand factor (rVWF) treatment for perioperative management of bleeding in adults (age 18 and older) with von Willebrand disease (VWD).1 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.1,3-4

"The expanded approval of VONVENDI represents a new treatment option for the surgical setting that can be tailored to each patient's individual needs,"1,3-5 said Andreas Busch, Global Head of Research and Development, Shire. "It's an important milestone in support of our vision of personalizing treatment and helping to address unmet needs for people with bleeding disorders."1,3-5

People with VWD lack proper quantities of VWF or functioning VWF, and they may or may not have a secondary factor VIII (FVIII) deficiency.5 Since not every person with VWD or every bleed requires FVIII replacement, VONVENDI allows healthcare providers to dose recombinant VWF independent of recombinant FVIII based on clinical judgement for each patient, taking into account severity, site of bleeding, the patient's medical history and monitoring of appropriate clinical and laboratory measures.1 Independent dosing with VONVENDI offers an individualized approach to bleed control in appropriate patients undergoing surgery.1

"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, M.D., Professor of Pediatrics and Medicine, University of Illinois College of Medicine, and Medical Director and President, 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.1,7 The expanded use for 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."1,5

The approval of VONVENDI in surgical settings was based on results from a Phase 3 prospective, open-label, multicenter trial to evaluate the efficacy and safety of VONVENDI with or without recombinant FVIII treatment in elective surgical procedures in adults (age 18 years and older) diagnosed with severe VWD.1 Results from the study 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.1 The overall median dosing frequency of once-daily was demonstrated to normalize hemostasis in appropriate patients.1 One study participant developed deep vein thrombosis three days after undergoing hip replacement surgery while receiving VONVENDI.1

In addition to the expanded use of VONVENDI, the updated Prescribing Information includes new information about pharmacokinetics and storage of VONVENDI. VONVENDI can be stored at refrigerated temperature 2°C to 8°C (36°F to 46°F) or room temperature not to exceed 30°C (86°F). Do not freeze. Store VONVENDI in the original box and protect it from extreme exposure to light.

VONVENDI was first approved by the FDA in December 2015.