Vonvendi Approved by FDA to Treat Common Bleeding Disorder

Vonvendi Approved by FDA to Treat Common Bleeding Disorder
December 9, 2015

For individuals suffering from von Willebrand disease (VWD), a new treatment option will soon be available.

The Food and Drug Administration (FDA) has announced its approval for Vonvendi, von Willebrand factor (Recombinant), for use in adults 18 years of age and older. Vonvendi is the first FDA-approved recombinant von Willebrand factor, and is approved for the on-demand treatment and control of bleeding episodes in adults diagnosed with VWD.

"Patients with heritable bleeding disorders should meet with their health care provider to discuss appropriate measures to reduce blood loss," said Karen Midthun, director of the FDA's Center for Biologics Evaluation and Research. "The approval of Vonvendi provides an additional therapeutic option for the treatment of bleeding episodes in patients with von Willebrand disease."

VWD is the most common inherited bleeding disorder, affecting approximately 1 percent of the US population. VWD is caused by a deficiency or defect in von Willebrand factor, a protein that is critical for normal blood clotting. Patients with VWD can develop 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 of 69 adult participants with VWD. These trials demonstrated that Vonvendi was safe and effective for the on-demand treatment and control of bleeding episodes from a variety of different sites in the body. No safety concerns were identified in the trials.

The FDA granted Vonvendi orphan product designation for these uses. Orphan product designation is given to drugs intended to treat rare diseases in order to promote their development.