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FDA Expands Approval for Recombinant VWF, Includes Pediatric Patients
FDA expands Vonvendi approval for all von Willebrand disease types, enhancing treatment options for adults and children with this rare condition.
The US Food and Drug Administration (FDA) has expanded the approval of recombinant von Willebrand factor (VWF), marketed as Vonvendi, to include routine preventative 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.
Announced on September 05, 2025, 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 Type 3 VWD. According to the FDA announcement, the therapy is the only non-plasma-derived VWF product approved for VWD in the US.
“This approval demonstrates FDA flexibility in evaluating applications of therapeutics to treat rare diseases,” said Vinay Prasad, MD, MPH, director of the FDA’s Center for Biologics Evaluation and Research. “When we see the trifecta of plausible mechanism of action, robust pharmacology/biologic science, and supportive clinical study data, we promptly act even if that data is derived from a small sample size study.”
Prior to the September 05, 2025, approval of recombinant VWF, the agent had received 3 prior approvals in the past decade. The initial approval for recombinant VWF occurred in December 2015, when the FDA approved the therapy for treatment of bleeding episodes in patients with VWD based on a pair of clinical trials including 69 adult participants.
In 2018, the FDA approved the treatment for perioperative management of bleeding in adults with VWD based on data from a phase 3 prospective, open-label, multicenter trial assessing use with or without recombinant FVIII treatment in elective surgical procedures in adults with severe VWD. In 2022, it received approval for routine prophylaxis to reduce the frequency of bleeding episodes in adults with severe Type 3 VWD receiving on-demand therapy.
“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 trial investigator Jonathan C. Roberts, MD, 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. “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.”
According to a release from Takeda Pharmaceuticals, the September 2025 approval is supported by on data from a trio of clinical trials, including a phase 3 study in children with VWD and a phase 3b 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 non-surgical bleeds in clinical trials treated with a single infusion in children and adults.
“Today’s approval is a testament to the collaborative interactions between CBER review teams and product developers, aimed at making innovative therapies available for use in pediatric populations, at the same time as adults,” said Vijay Kumar, MD, acting director of the CBER Office of Therapeutic Products.
