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FDA approves Vonvendi to treat bleeding episodes
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The FDA approved von Willebrand factor, recombinant, for use in adults who have von Willebrand disease.
Von Willebrand factor, recombinant (Vonvendi, Baxalta) is the first recombinant von Willebrand factor to receive FDA approval. It is indicated for on-demand treatment and control of bleeding episodes.
Von Willebrand disease is an inherited bleeding disorder caused by a deficiency or defect in von Willebrand factor, a protein that is crucial for normal blood clotting. The condition — which affects about 1% of the U.S. population, according to the FDA — can lead to severe bleeding from the intestines, gums and nose, as well as bleeding into joints and muscles.
The FDA based its decision on results of two clinical trials that included 69 adults. The trials showed Vonvendi was effective and safe for on-demand treatment and control of bleeding episodes at various sites in the body.
Researchers identified no safety concerns in the trials, according to the FDA. The most common adverse reaction associated with Vonvendi was pruritus.
“Patients with heritable bleeding disorders should meet with their health care provider to discuss appropriate measures to reduce blood loss,” Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “The approval of Vonvendi provides an additional therapeutic option for the treatment of bleeding episodes in patients with von Willebrand disease.”
The FDA previously granted orphan drug designation to Vonvendi for these indications.