FDA Expands Approval for Recombinant VWF

The U.S. Food and Drug Administration (FDA) has expanded the approval of recombinant von Willebrand factor (VWF), marketed as Vonvendi, to include routine preventive use in adults with all types of von Willebrand disease (VWD) and on-demand treatment of bleeding episodes and perioperative use in children with VWD.

The approval makes recombinant VWF the first non-plasma-derived VWF product available for pediatric populations and expands the previous indications for recombinant VWF, which were for on-demand treatment of bleeding episodes and perioperative use in adults and preventative use only in adults with rype 3 VWD. According to FDA, the therapy is the only non-plasma-derived VWF product approved for VWD in the U.S.

Approval is supported by data from a trio of clinical trials, including a Phase III study in children with VWD and a Phase IIIb continuation trial in adults and children with VWD, in addition to supportive real-world data. The company pointed out recombinant VWF has a uniquely long half-life of 22.6 hours for adults and 14.3 hours for children, with most nonsurgical bleeds in clinical trials treated with a single infusion in children and adults.

Prior to this expanded approval, Vovendi had received three prior approvals in the past decade. The initial approval for recombinant VWF occurred in December 2015, when FDA approved the therapy for treatment of bleeding episodes in patients with VWD based on a pair of clinical trials, including 69 adult participants.