FDA Approves Expanded Use of Takeda VONVENDI for Adults, Children with Von Willebrand Disease

The U.S. Food and Drug Administration (FDA) has approved an expanded indication for Takeda's VONVENDI, a recombinant von Willebrand factor (VWF) replacement therapy, to include routine prophylaxis for adults with all types of von Willebrand Disease (VWD) and on-demand and perioperative management of bleeding in pediatric patients. VONVENDI is now the only recombinant VWF therapy approved for both adults and children with VWD, a common bleeding disorder affecting over 3 million people in the U.S. Clinical trials demonstrated that most bleeding episodes in both adults and children were effectively treated with a single infusion, highlighting the therapy's efficacy and convenience. This expanded approval marks a significant advancement in treatment options for VWD patients, potentially improving quality of life by reducing bleeding frequency and providing broader access to prophylactic care. While generally well-tolerated, some adverse reactions such as headache, nausea, and vomiting were reported in clinical studies. Takeda's approval strengthens its position in the hematology market and underscores ongoing efforts to innovate treatments for rare diseases.