Recombinant Treatment Drug Approval Sought for von Willebrand Disease

March 30, 2015
Rachel Lutz

Hemophilia Reports, March 2015, Volume 2, Issue 1

Producers of a von Willebrand factor recombinant treatment are seeking a biologics license after already being granted orphan drug status by the FDA.

Baxter submitted an application for US Food and Drug Administration (FDA) approval for an investigational recombinant treatment for von Willebrand Disease, the company announced.

The company submitted a biologics license application for the approval of BAX111, the first highly purified recombinant von Willebrand Factor (VWF) treatment in clinical development. It is intended for von Willebrand disease patients and is based on the results of a phase 3 clinical trial which assessed the drug’s safety, efficacy, and pharmacokinetics.

Von Willebrand disease an autosomal genetic disorder related to quantitative deficits and/or qualitative defects of VWF, which results in impaired homeostasis. It is also the most common inherited bleeding disorder, affecting 1 to 2 percent of the general population. Most often, von Willebrand disease patients experience mild symptoms, but some patients experience severe bleeding events similar to hemophilia patients’ bleeding episodes.

To evaluate BAX111, researchers observed 22 patients who were treated with 100 percent success based on a 4 point efficacy rating scale. The majority of bleeding events (81.1 percent) were resolved with a single transfusion. The researchers observed 125 adverse events after 318 infusions occurred in 25 out of 37 subjects. There were 8 adverse events that researchers determined were causally related to BAX111: 6 non serious related adverse events (tachycardia, infusion site paresthesia, electrocardiography T wave inversion, dysgeusia, generalized pruritis, and hot flush) occurred in 4 patients, and 2 related serious adverse events (chest discomfort and increased heart rate) occurred in 1 patient.

“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 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.”

Additional information about the study will be published in the coming months. The European Commission and the FDA have both already granted orphan drug designation for BAX111.