Baxter Submits Application for FDA Approval of Von Willebrand Disease Treatment

Baxter International Inc has submitted a biologics license application to the FDA for the approval of BAX111, the first highly purified recombinant von Willebrand factor in clinical development as a treatment for von Willebrand disease, the company announced in a press release.

Von Willebrand disease is an autosomal genetic disorder related to quantitative deficits and/or qualitative defects of von Willebrand factor that result in impaired hemostasis. While many people with the disease only experience mild symptoms, some patients can experience severe bleeding episodes similar to those experienced by patients with hemophilia.

“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, in a press release. “Filing for US 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 application was filed following the completion of a Phase III, multicenter, open-label clinical trial assessing the safety, effectiveness, and pharmacokinetics of BAX111. The study met its primary efficacy end point, which was defined by the number of patients who achieved treatment success for control of bleeding episodes. All 22 patients who were treated in the full analysis set experienced a 100% treatment success rating based on a 4-point efficacy rating scale that compared the 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, with 81.1% of events resolving after a single infusion.

Following 318 infusions, 25 out of 37 patients reported a total of 125 adverse events. Eight of these events were determined to be causally related to BAX111, including 6 nonserious events (tachycardia, infusion site paresthesia, electrocardiography T-wave inversion, dysgeusia, generalized pruritus, and hot flush) that occurred in 4 patients, and 2 serious events (chest discomfort and increased heart rate) experienced by 1 patient.

Baxter plans to publish additional data from the trial in the coming months. Both the FDA and the European Commission have designated BAX111 as an orphan drug.