Bleeding disorder drug shows positive results

After positive phase 3 trial data for its treatment for the bleeding disorder von Willebrand disease, Baxalta Inc. is awaiting FDA approval for the drug.

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Published in a recent issue of Blood, the Journal of theAmerican Society of Hematology, the study found that 100% of the patients treated with BAX 111, a highly purified recombinant von Willebrand factor (rVWF) candidate, achieved success in the management of bleeding episodes.

If approved, BAX 111 would become the first recombinant replacement treatment for managing bleeding episodes for von Willebrand patients. Both FDA and the European Medicines Agency granted orphan drug designation for BAX 111 in November 2010.

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“Von Willebrand disease is the most common hereditary bleeding disorder, yet few treatment options exist,” said John Orloff, MD, head of research & development and chief scientific officer, Baxalta. “BAX 111 has the potential to transform the standard of care, especially for patients with severe von Willebrand disease, by offering an effective individualized treatment option.”

There is no cure for von Willebrand disease and the primary treatments for severe von Willebrand disease are transfusion therapy and plasma-derived VWF concentrates, according to Baxalta.

The phase 3 multicenter, open-label clinical trial assessed the safety, efficacy and pharmacokinetics of BAX 111 in the on-demand treatment of 37 patients with severe von Willebrand disease at trial sites in the United States, Europe, Australia, Japan, Russia and India.

All study participants (100%) reported a mean efficacy rating of <2.5 on a 4-point scale where 1 represented excellent control and 4 was no bleeding control. One infusion was sufficient to control 81.8% of bleeds, with a median of 2 infusions required to treat major bleeds.

The most common adverse events to BAX 111 were headache, vomiting/nausea and anemia (iron deficiency anemia), which were not considered to be related to treatment. One patient experienced 2 simultaneous serious adverse events related to treatment, characterized by chest discomfort and increased heart rate during infusion, which were resolved within 3 hours without further symptoms observed.

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