FDA Approves Expanded Use of Takeda’s Vonvendi

Rare Daily Staff

The U.S. Food and Drug Administration approved the expanded use of Takeda’s Vonvendi to include routine prophylaxis to reduce the frequency of bleeding episodes in adults with type 1 and type 2 von Willebrand disease.

Vonvendi was previously approved for on-demand and perioperative use in adults with von Willebrand disease (VWD), and for routine prophylactic use in adults with severe type 3 VWD receiving on-demand therapy.

VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the U.S. population. The condition is caused by a deficiency or dysfunction of von Willebrand factor (VWF), one of several proteins in the blood needed to facilitate proper clotting. Because of this defect or deficiency, blood is not able to clot effectively in people with VWD, which may result in heavy menstrual periods, easy bruising, or frequent nosebleeds. Bleeding caused by VWD varies greatly among patients. The disease can lead to life-threatening bleeding from injuries or procedures such as surgery, dental work, or childbirth, as well as prolonged nosebleeds, heavy menstrual bleeding, and easy bruising, all of which can affect quality of life.

Vonvendi is a recombinant von Willebrand factor (rVWF) treatment for adults and children with VWD. It is the only recombinant VWF product approved for VWD in the United States and the first approved for pediatric patients. Prior to this approval, only plasma-derived VWF products were available to children.

The approval is based on data from three clinical trials: a phase 3 trial in adults with VWD, a phase 3 study in children with VWD, and a phase 3b continuation trial in adults and children with VWD, as well as supportive real-world data.

Takeda has submitted applications seeking approval to expand the indications of Vonvendi for on-demand and perioperative management of bleeding in pediatric patients with VWD in Japan, and of Veyvoni, as it is marketed in Europe, for on-demand use in pediatric patients with VWD in the European Union.

“The FDA’s approval of Vonvendi for routine prophylactic use in adults with all VWD types marks a pivotal milestone in advancing care for this challenging condition. Prophylactic treatment is critical to reduce the frequency of bleeds in adults, including spontaneous and joint bleeds,” said Jonathan Roberts, associate medical and research director at the Bleeding and Clotting Disorders Institute, associate professor of pediatrics and medicine at the University of Illinois College of Medicine at Peoria, and an investigator in the Vonvendi prophylaxis study. “Establishing prophylaxis as the standard of care for all adults with VWD, regardless of subtype, represents a significant step forward in helping to manage bleeding risk and the burdens that bleeds can have on patients’ daily lives.”