FDA Approves Recombinant Therapy for Patients with von Willebrand Disease

The US Food and Drug Administration (FDA) has approved von Willebrand factor (recombinant) (VONVENDI), a recombinant von Willebrand factor (rVWF) treatment for perioperative management of bleeding in adults with von Willebrand disease.

The approval in surgical settings was based on results from a phase 3 prospective, open-label trial that evaluated the efficacy and safety of VONVENDI with or without recombinant factor VIII treatment in elective surgical procedures in adults diagnosed with severe von Willebrand disease.

“The expanded use for VONVENDI in surgical settings gives health care professionals flexibility in treating von Willebrand disease with an appropriate dose of von Willebrand factor, with or without recombinant factor VII, based on each patient’s unique needs,” Michael Tarantino, MD, professor of pediatrics and medicine, University of Illinois College of Medicine, medical director and president, The Bleeding and Clotting Disorders Institute, said in a statement.

Study findings demonstrated that VONVENDI met its primary endpoint, which included overall hemostatic efficacy assessed 24 hours after the last perioperative VONVENDI infusion or at completion of study visit — whichever occurred first.

Median dosing frequency occurred once-daily and was demonstrated to normalize hemostasis in patients.

One patient developed deep vein thrombosis 3 days after undergoing hip replacement surgery while receiving treatment with VONVENDI.

In addition to the expanded indication, VONVENDI is also indicated for use in adults diagnosed with von Willebrand disease for on-demand treatment and control of bleeding episodes. It is not recommended for use in patients who have experienced life-threatening hypersensitivity reactions to VONVENDI or its components.

The updated prescribing information now includes new information about pharmacokinetics and storage of VONVENDI which can be stored at refrigerated temperature 2°C to 8°C (36°F to 46°F) or room temperature and should not exceed 30°C (86°F).

“Persons with von Willebrand disease face a heightened risk of bleeding during surgery and may require factor treatment before, during or after surgery,” Tarantino concluded. “For surgeries requiring repeated, frequent infusions with combined von Willebrand factor and factor VII concentrates, an excessive rise in factor VII levels may increase the risk of thromboembolic complications, such as blood clots.”

The most common adverse effects observed in greater than or equal to 2% of subjects (n=80) were generalized pruritus, nausea and dizziness.

VONVENDI is the first and only recombinant treatment for adults living with von Willebrand disease and was first approved in December 2015 by the FDA.