Von Willebrand Disease

August 17, 2015

Baxalta has released results of a phase III study of BAX 111 for Von Willebrand disease. The phase III multicenter, open-label clinical trial assessed the safety, efficacy and pharmacokinetics of BAX 111 in the on-demand treatment of 37 patients with severe von Willebrand disease at trial sites in the U.S., Europe, Australia, Japan, Russia and India. The primary endpoint was the number of patients experiencing successful treatment for bleeding episodes. Secondary endpoints included additional efficacy measures, pharmacokinetics, the number of infusions and the number of units administered per bleeding episode. All study participants reported a mean efficacy rating of <2.5 on a fourpoint scale where one equaled excellent control and four equaled no bleeding control. Data also indicated that one infusion was sufficient to control 81.8% of bleeds, with a median of two infusions required to treat major bleeds. No thrombotic events or severe allergic reactions were observed, and none of the participants developed anti-von Willebrand factor (VWF) binding or neutralizing antibodies to VWF. The most common adverse events in the study were headache, vomiting/nausea and anemia (iron deficiency anemia), which were not considered to be related to treatment. Both the FDA and the EMA granted Orphan Drug designation in November 2010. BAX 111 currently is under review by the FDA.

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