Baxter files BLA at FDA for BAX 111 for von Willebrand Disease.

Baxter International Inc. announced that the company has submitted a biologics license application (BLA) to the FDA for the approval of BAX 111, 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.

The filing was based on the completion of a Phase III, multi-center, open-label clinical trial assessing the safety, efficacy and pharmacokinetics of BAX 111. 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.