HOBOKEN, N.J. The Food and Drug Administration has given orphan drug designation to a treatment for an inherited bleeding disorder that occurs in fewer than 1-in-1,000 people.
Octapharma USA announced that the FDA had given the designation to the drug Wilate (von Willebrand factor/factor VIII concentrate, human), a treatment for von Willebrand disease. The FDA grants orphan drug designation to treatments for rare diseases.
Von Willebrand disease causes bleeding episodes in patients, either spontaneously or as a result of trauma.