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FDA Approves First Recombinant von Willebrand Factor to Treat Bleeding
The FDA has approved Vonvendi (von Willebrand factor [recombinant], Baxalta U.S., Inc.) for use in adults (18 years of age and older) who have von Willebrand disease (VWD). Vonvendi is the first FDA-approved recombinant von Willebrand factor. It is approved for the on-demand (as-needed) treatment and control of bleeding episodes in adults diagnosed with VWD.
VWD is the most common inherited bleeding disorder, affecting approximately 1% of the U.S. population. Men and women are equally affected by the disease, which is caused by a deficiency or defect in von Willebrand factor, a protein that is critical for normal blood clotting. Patients with VWD can experience severe bleeding from the nose, gums, and intestines, as well as into muscles and joints. Women with VWD may have heavy menstrual periods lasting longer than average and may experience excessive bleeding after childbirth.
The safety and efficacy of Vonvendi were evaluated in two clinical trials involving a total of 69 adult participants with VWD. These studies demonstrated that Vonvendi was safe and effective for the on-demand treatment and control of bleeding episodes from a variety of sites in the body. No safety concerns were identified. The most common adverse event was generalized pruritus.
The FDA had granted Vonvendi orphan drug status for these uses.
Source: FDA; December 8, 2015.