FDA Expands Approval of Recombinant von Willebrand Factor

The Food and Drug Administration recently approved the expanded use of Takeda’s recombinant von Willebrand factor (Vonvendi) in adults and children with von Willebrand disease (VWD).

The approval allows for prophylactic use in adults with all types of VWD, and for on-demand treatment of bleeding episodes and perioperative management in pediatric patients. The therapy is the only recombinant, non-plasma-derived VWF product approved in the U.S. It is also the first recombinant VWF therapy available for pediatric patients.

FDA previously approved the therapy for on-demand treatment and perioperative use in adults. Prophylactic use in adults was limited to patients with type 3 VWD, its most severe form. Only plasma-derived products were available for pediatric patients.