Drugs Xagena
The FDA ( Food and Drug Administration ) has approved Vonvendi, von Willebrand factor ( recombinant ), 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, and is approved for the on-demand ( as needed ) treatment and control of bleeding episodes in adults diagnosed with von Willebrand disease.
von Willebrand disease is the most common inherited bleeding disorder, affecting approximately 1% of the U.S. population. Men and women are equally affected by von Willebrand disease, which is caused by a deficiency or defect in von Willebrand factor, a protein that is critical for normal blood clotting.
Patients with von Willebrand disease can develop severe bleeding from the nose, gums, and intestines, as well as into muscles and joints.
Women with von Willebrand disease 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 von Willebrand disease. 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 most common adverse reaction observed was generalized itching.
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. ( Xagena )
Source: FDA, 2015
XagenaMedicine_2015
