Recombinant Treatment for von Willebrand Disease Meets Primary Efficacy Endpoint in Phase III Trial
NDA submission expected before end of year
Positive results have been reported from a phase III clinical trial evaluating the safety, efficacy, and pharmacokinetics of BAX 111 (Baxter International), a recombinant von Willebrand factor under investigation for the treatment of bleeding episodes in patients with von Willebrand disease, the most common type of inherited bleeding disorder.
BAX 111 is the first recombinant treatment in clinical development for this condition.
The phase III, open-label study met its primary efficacy endpoint, as all patients achieved pre-specified success in the on-demand treatment of bleeding events (22 of 22 patients who experienced bleeds during the study).
BAX 111 was administered with Advate (antihemophilic factor [recombinant], plasma/albumin-free method, Baxter) or as a stand-alone therapeutic agent in the on-demand treatment of 37 patients with severe von Willebrand disease in the U.S., Europe, Australia, Japan, Russia, and India. The study’s primary endpoint was the number of patients experiencing successful treatment of bleeding episodes. Secondary endpoints included additional efficacy and safety measures, pharmacokinetics, and health-related quality of life.
There were no reports of inhibitor development or thrombotic events in the study participants. The most common adverse events (AEs) were headache, vomiting, nausea, and iron deficiency anemia, which were not considered to be related to treatment. One serious AE was related to treatment; this AE was characterized by chest discomfort and increased heart rate during infusion, which rapidly resolved without further complications.
The FDA granted an orphan drug designation for BAX 111 in November 2010. A new drug application (NDA) is expected before the end of 2014.
Source: Baxter International; April 16, 2014.