Recombinant Therapy for von Willebrand Disease Approved

This morning, Shire announced the expanded U.S. Food and Drug Administration (FDA) approval of Vonvendi, a recombinant von Willebrand factor (rVWF), for the treatment of bleeding in adults with von Willebrand disease in surgical settings.

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.

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

“The expanded approval of Vonvendi represents a new treatment option for the surgical setting that can be tailored to each patient’s individual needs,” said Andreas Busch, Global Head of Research and Development at Shire in a press release. “It’s an important milestone in support of our vision of personalizing treatment and helping to address unmet needs for people with bleeding disorders.”

Study results found that Vonvendi met its primary endpoint, exhibiting overall hemostatic efficacy assessed 24 hours after the last perioperative infusion or at completion of study visit — whichever transpired sooner.

Overall median dosing frequency of once-daily was demonstrated to normalize hemostasis in patients, and it was noted that only 1 study participant developed deep vein thrombosis 3 days after undergoing hip replacement surgery while being administered the treatment.

Intra- and postoperative hemostasis achieved with recombinant VWF with or without recombinant FVIII was rated as “excellent,” defined as good as or better than expected; or “good,” defined as probably as good as expected for all 15 study participants treated with Vonvendi. To consider the study to be a success, a rating of excellent or good was required.

“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.

In addition to the expanded use of Vonvendi, the updated Prescribing Information includes new information about pharmacokinetics and storage of it. 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).

“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 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,” Dr. Tarentino continued.

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

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