New Rx Treats Most Common Inherited Bleeding Disorder

Vonvendi, von Willebrand factor (Recombinant), FDA-approved to treat von Willebrand disease

/ Author:  / Reviewed by: Jennifer Gershman, PharmD, CPh

(RxWiki News) Patients with von Willebrand disease (VWD) lack a protein that's essential for healthy blood clotting. But a newly approved medication could help with that.

That new medication is Vonvendi, von Willebrand factor (Recombinant). The US Food and Drug Administration (FDA) approved it Tuesday to treat adults with VWD. The drug is approved to control bleeding episodes in VWD patients.

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

Among inherited bleeding disorders, VWD is the most common. According to the FDA, it affects about 1 percent of people in the US. These patients either do not have von Willebrand factor — a protein needed to make the blood clot — or their von Willebrand factor is somehow defective.

When the blood can't clot correctly, it's more likely to bleed out excessively. VWD patients can have episodes of severe bleeding, which can be external or internal.

But Vonvendi could help with these dangerous effects of VWD. The FDA approved this Baxalta US drug after it appeared safe and effective in two clinical trials of 69 total patients. In fact, these studies uncovered no safety concerns tied to the drug.

"The most common adverse reaction observed was generalized pruritus (itching)," according to an FDA press release.

Review Date: 
December 8, 2015
Last Updated:
December 9, 2015