Baxter International Inc. today announced that the company has submitted a biologics license application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for the approval of BAX111, the first highly-purified recombinant von Willebrand Factor (rVWF) in clinical development as a treatment for patients with von Willebrand disease, the most common type of inherited bleeding disorder.
”If approved, BAX111 will be the first recombinant replacement treatment for von Willebrand disease, offering an important new option that may provide greater flexibility in treating patients with this challenging disease,” said John Orloff, vice president and global head of research and development at Baxter BioScience. ”Filing for U.S. approval for this treatment helps us further advance our pursuit of new treatment options and improved quality of care for people with a range of bleeding disorders around the world.”
The filing was based on the completion of a Phase III, multi-center, open-label clinical trial assessing the safety, efficacy and pharmacokinetics of BAX111. The study met its primary efficacy endpoint defined by the number of patients who achieved treatment success for control of bleeding episodes. All patients treated in the full analysis set (n=22) experienced a 100% treatment success rating based on a 4-point efficacy rating scale, comparing prospectively estimated number of infusions needed to treat the bleeding episodes to the actual number of infusions administered. The median number of infusions required to treat bleeding events in the trial was 1.0 and the majority of events (81.1%) were resolved with a single infusion.
A total of 125 adverse events (AE’s) following 318 infusions occurred in 25/37 subjects. Eight AEs were considered causally related to BAX111: six non-serious related AEs (tachycardia, infusion site paresthesia, electrocardiography (ECG) T-wave inversion, dysgeusia, generalized pruritis and hot flush) occurred in four patients, and two related SAEs (chest discomfort and increased heart rate) occurred in one patient.
Baxter expects to publish additional data from the trial in the coming months. Both the European Commission and the U.S. Food and Drug Administration have granted orphan-drug designation for BAX111.
About von Willebrand Disease
Von Willebrand Disease (VWD) is an autosomal genetic disorder related to quantitative deficits and/or qualitative defects of VWF, the result of which is impaired hemostasis. It is the most common hereditary coagulation disorder, occurring in approximately one to two percent of the general population. Many people who have VWD may experience mild symptoms, but some patients can experience severe bleeding events similar to bleeding experienced by patients with hemophilia.