In April, Baxter International, Inc., announced results from a Phase 3, multicenter clinical trial evaluating the safety, efficacy and pharmacokinetics (PK) of BAX 111, the company’s recombinant von Willebrand factor (rVWF) product to treat bleeding episodes in patients with von Willebrand disease (VWD). It is the first recombinant therapy for VWD to reach this stage of development.
In the trial, BAX 111 was administered together with ADVATE (Baxter’s recombinant factor VIII product) or as a stand-alone therapeutic agent in the on-demand treatment of 37 patients with severe VWD at trial sites in Europe, Australia, Japan, Russia, India and the US. The primary endpoint was the number of patients experiencing successful treatment for bleeding episodes. Secondary endpoints included additional efficacy and safety measures, pharmacokinetics (measurement of the presence of a drug in the body over time) and health-related quality of life.
According to a Baxter press release, the trial met its “primary efficacy endpoint,” as 22 participants who experienced bleeds during the study achieved success using on-demand treatment with BAX 111.
There were no reports of inhibitor development or thrombotic events in the subjects. The most common adverse events in the study were headache, vomiting/nausea and iron deficiency anemia, which were not considered to be related to treatment. There was one serious adverse event related to treatment, characterized by chest discomfort and increased heart rate during infusion, which rapidly resolved without further complication.
“As the first recombinant, stand-alone treatment in development, BAX 111 has the potential to offer people with von Willebrand disease a new therapeutic option that may allow for greater precision and flexibility in managing the disease,” said Bruce Ewenstein, MD, PhD, vice president of clinical affairs, in Baxter BioScience. “With these findings, we have taken another significant step forward as we continue to expand on our increasingly broad pipeline of potential treatments to improve outcomes for patients with a range of bleeding disorders.”
Source: Baxter press release dated April 16, 2014
