FDA Approves Expanded Use of Vonvendi for All Types of Von Willebrand Disease

The U.S. Food and Drug Administration (FDA) has officially authorized a broader application of Vonvendi (von Willebrand factor [recombinant]) for patients with von Willebrand disease (VWD), a genetic bleeding disorder affecting blood clotting. This new approval allows for its use in routine preventive care in adults with all types of VWD, as well as on-demand treatment for bleeding episodes and perioperative management for children suffering from the condition.

Previously, Vonvendi was approved only for treating bleeding episodes and perioperative procedures in adults, and as a preventive measure only for adults with severe type 3 VWD. Now, it is the only non-plasma-derived von Willebrand factor product approved in the U.S. for a wider range of VWD patients, including children, marking a significant advancement in treatment options.

This approval was granted under a priority review process and was designated as an orphan drug, emphasizing its importance for rare disease management. Multiple clinical studies have demonstrated Vonvendi's effectiveness in controlling bleeding episodes and managing surgeries across all age groups with VWD. Common side effects reported in clinical trials include headache, nausea, vomiting, dizziness, and skin itchiness.

Vinay Prasad, M.D., director at the FDA's Center for Biologics Evaluation and Research, highlighted the agency's flexibility in approving therapies for rare diseases, especially when supported by scientific evidence and clinical data. The new approval underscores the ongoing efforts to improve treatment for patients with challenging bleeding disorders.

This advancement demonstrates a major step forward in managing VWD effectively, offering new hope for patients and families affected by this hereditary condition.