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FDA expands approval of Vonvendi for von Willebrand disease
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The FDA expanded the approval of Vonvendi for the treatment of bleeding in adults with von Willebrand disease to include surgical settings, according to the agent’s manufacturer.
Vonvendi (von Willebrand factor [recombinant], Shire) — approved by the FDA in 2015 — is the only recombinant treatment for adults with von Willebrand disease, the most common inherited bleeding disorder. In addition to this new indication for the perioperative management of bleeding in adults, Vonvendi is indicated for on-demand treatment and control of bleeding episodes.
“The expanded approval of Vonvendi represents a new treatment option for the surgical setting that can be tailored to each patient’s individual needs,” Andreas Busch, global head of research and development at Shire, said in a company-issued 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.”
The FDA based this decision on data from a phase 3 open-label multicenter trial that evaluated the efficacy and safety of Vonvendi with or without recombinant factor VIII treatment in elective surgical procedures among adults with severe von Willebrand disease.
The study met its primary endpoint, demonstrating overall hemostatic efficacy 24 hours after the last perioperative infusion or at completion of study visit.
The median dosing frequency of once daily demonstrated normalization of hemostasis in appropriate patients.
One patient developed deep vein thrombosis 3 days after undoing hip replacement surgery while on Vonvendi.
Patients with von Willebrand disease are deficient in or lack functioning von Willebrand factor, and they may have secondary factor VIII deficiency. Because not every patient requires factor VIII deficiency, Vonvendi allows health care providers to dose recombinant von Willebrand factor independently of recombinant factor VIII according to the severity and site of bleeding and the patient’s medical history, according to the release.
“Persons with von Willebrand disease face a heightened risk [for] bleeding during surgery and may require factor treatment before, during or after surgery,” Michael Tarantino, MD, professor of pediatrics and medicine at University of Illinois College of Medicine and medical director and president of The Bleeding and Clotting Disorders Institute, said in the release. “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 [for] thromboembolic complications, such as blood clots. 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 VIII, based on each patient’s unique needs.”