The U.S. Food and Drug Administration (FDA) has expanded the approved use of Vonvendi (von Willebrand factor [recombinant]) to include routine prophylaxis in adults with all forms of von Willebrand disease (VWD), a rare genetic bleeding disorder.
The therapy is also now authorized for on-demand treatment of bleeding episodes and for perioperative use in children with VWD.
The decision, made under priority review with orphan drug designation, significantly extends Vonvendi’s reach. Until now, the product was limited to on-demand and perioperative use in adults, and for preventive treatment only in those with type 3 VWD, the most severe form.
With this expansion, Vonvendi becomes the first recombinant von Willebrand factor therapy available for U.S. pediatric patients and remains the only non–plasma-derived option approved nationwide.
Clinical studies demonstrated Vonvendi’s effectiveness in managing bleeding episodes, supporting surgical care, and reducing bleeding risk in adults. Reported side effects in at least 2% of participants included headache, nausea, vomiting, dizziness, and itching.
“This approval demonstrates FDA flexibility in evaluating therapeutics for rare diseases,” said Vinay Prasad, M.D., director of the FDA’s Center for Biologics Evaluation and Research.
“When we see the trifecta of plausible mechanism, robust science, and supportive clinical data, we act quickly—even when the evidence comes from smaller trials.”
The expanded approval was granted to Takeda, Vonvendi’s manufacturer.
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