Baxalta receives FDA approval for von Willebrand disease treatment
VWD is the most commonly diagnosed inherited bleeding disorder, affecting approximately one percent of U.S. citizens. Although an equal number of cases have been diagnosed in men and women, women with VWD can have more frequent severe bleeding episodes due to menstruation and childbirth.
“The approval of Vonvendi provides an additional therapeutic option for the treatment of bleeding episodes in patients with von Willebrand disease,” Dr. Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research, said.
The areas of the body most often affected by VWD bleeding are the nose, gums, intestines, muscles and joints.
Vonvendi, the first drug approved by the FDA for treatment of von Willebrand disease, is only recommended for use by patients who are 18 or older. Its safety and performance were tested with two clinical trials using 69 VMD patients as subjects.
Vonvendi is to be administered when bleeding events occur. The FDA has granted orphan drug designation for Vonvendi -- a designation reserved for therapies used to treat extremely rare diseases.
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