MONDAY, Sept. 8, 2025 (HealthDay News) -- The U.S. Food and Drug Administration has approved expanded use of Vonvendi (von Willebrand factor [recombinant]) for routine prophylactic use in adults with all types of the blood-clotting disorder von Willebrand disease (VWD), as well as for on-demand treatment of bleeding episodes and perioperative use in children with VWD.
This approval was completed under priority review with orphan drug designation. Vonvendi previously was approved only for on-demand treatment of bleeding episodes and perioperative use in adults and preventative use only in adults with the most serious type of VWD, type 3. Now, Vonvendi is the only non-plasma-derived von Willebrand factor product approved for VWD in the United States and is the first recombinant von Willebrand factor product approved for U.S. pediatric patients.
Multiple clinical studies showed the effectiveness of Vonvendi in treatment of bleeding episodes and use in perioperative management in patients with VWD of all ages. It also showed success for prevention of bleeding episodes in adults with VWD. The most common adverse reactions (≥2 percent of patients in clinical trials) included headache, vomiting, nausea, dizziness, and itchy skin.
"This approval demonstrates FDA flexibility in evaluating applications of therapeutics to treat rare diseases," Vinay Prasad, M.D., director of the FDA Center for Biologics Evaluation and Research, said in a statement. "When we see the trifecta of plausible mechanism of action, robust pharmacology/biologic science, and supportive clinical study data, we promptly act even if that data is derived from a small sample size study."
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