Patient factors
Bleeding disorder patients tell FDA where they want drug therapies to improve
A dearth of options for patients who develop neutralizing antibodies against recombinant blood clotting factor therapies for bleeding disorders was one of the major concerns expressed by patient advocates last week at FDA's patient-focused drug development meeting for hemophilia A, hemophilia B, von Willebrand disease and other heritable bleeding disorders. The best option right now appears to be gene therapies, but the most advanced of these is most likely several years away from the market.
Normal clotting is mediated by interactions between platelets and clotting factors in the blood. When one or more clotting factors are deficient, bleeding disorders occur. Von Willebrand disease, caused by a deficiency of von Willebrand factor, is the most common bleeding disorder, followed by hemophilia A, which is caused by low levels of Factor VIII (FVIII) in the blood...