DEERFIELD (dpa-AFX) - Baxter International Inc. (BAX) reported topline results from a Phase 3 study, assessing the safety, efficacy and pharmacokinetics or PK of BAX 111, an investigational stand-alone recombinant treatment for von Willebrand Disease. The study of BAX 111, the first recombinant treatment in clinical development for this condition, achieved its primary efficacy endpoint, as all patients achieved pre-specified success in the on-demand treatment of bleeding events.
The Phase 3 multicenter, open-label trial evaluated the safety, efficacy and pharmacokinetics of BAX 111 administered along with ADVATE or as a stand alone therapeutic agent 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 study's primary endpoint was the number of patients experiencing successful treatment for bleeding episodes, while the secondary endpoints included additional efficacy and safety measures, pharmacokinetics and health-related quality of life or HRQoL.
The complete data from the trial, including efficacy and safety outcomes, are to be presented later in 2014. Both the European Commission and the U.S. Food and Drug Administration granted orphan-drug designation for BAX 111 in November 2010. The company plans to file for approval in the U.S. before this year end and, based on these results, intends to pursue a study of BAX 111 in a prophylaxis treatment setting before the year end.
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