On Tuesday, the US Food and Drug Administration approved the Baxalta biodrug Vonvendi to treat adults with the rare, inherited von Willebrand disease which can cause severe bleeding from the gums and the nose and even the intestines, and could also involve bleeding into the muscles and joints.
Indeed, it is a brutal condition, but those who suffered from this disease had no possible treatment options for many years.
“With no major therapeutic innovation in more than a decade, VONVENDI offers patients an important new option for VWD with a clinical profile that can help them manage this challenging chronic disease,” explains Joan Gill, MD, with the Blood Center and the Medical College of Wisconsin.
Gill is also the lead author of the recently concluded VONVENDI clinical study.
“Patients with heritable bleeding disorders should meet with their health care provider to discuss appropriate measures to reduce blood loss,” explains Karen Midthun, MD, who is the director of the US FDA’s Center for Biologics Evaluation and Research.
Of course, this new drug helps to add more options to these patients. According to the US Centers for Disease Control and Prevention (CDC), those who suffer from this disorder often have trouble with proper blood clotting.
Brian Goff is the executive vice president as well as President of Hematology at Baxalta—which is actually the biosciences arm of Baxter International—and he says, “With its unique feature of ultra-large multimers and the clinical profile seen in our pivotal study, VONVENDI is an important new option with the potential to redefine treatment for adults with von Willebrand disease.” He continues, “VONVENDI’s approval represents our commitment to advancing care as we continue to build a strong and differentiated portfolio that can help address challenging bleeding disorders for patients around the world.”