The US Food and Drug Administration (FDA) has expanded the indication for recombinant von Willebrand factor (VWF), marketed as Vonvendi, to include children with von Willebrand disease (VWD). The approved indication now permits the therapy to be prescribed to treat acute bleeding episodes in pediatric patients and to treat surgical patients.
The announcement, made on September 5, 2025, positions recombinant VWF as the first non–plasma-derived therapy available for pediatric cases of VWD in the United States. Previously, its use was confined to adults. Initial authorisation covered on-demand treatment of bleeding, which was later followed by approval for surgical use and, more recently, for routine prophylaxis in adults diagnosed with type 3 VWD.
“This approval highlights the FDA’s commitment to advancing treatment options for rare disorders, including for pediatric patients,” said Vinay Prasad, MD, MPH, director of the FDA’s Center for Biologics Evaluation and Research. “When we see the combination of a strong biologic rationale, sound pharmacology, and meaningful clinical evidence, we are able to act quickly, even when studies involve smaller patient groups.”
Prior to this latest decision, recombinant VWF had already received three FDA authorisations over the past decade. The first, granted in December 2015, was based on two clinical trials involving 69 adult patients and approved its use in treating bleeding episodes. In 2018, the use was extended to perioperative management of bleeding in adults by a multicenter phase 3 trial that evaluated its use with or without recombinant FVIII for elective procedures. A further step came in 2022, when prophylactic administration was approved for adults with severe type 3 VWD requiring on-demand therapy.
Takeda Pharmaceuticals reported that the pediatric approval is underpinned by evidence from three clinical investigations, including a phase 3 study designed specifically for children and a phase 3b continuation study that enrolled both adult and pediatric participants. In addition, real-world data strengthened the case for the expanded indication. Findings revealed that most non-surgical bleeds in children could be effectively controlled with a single infusion. Pharmacokinetic data showed a half-life of 14.3 hours in pediatric patients, compared with 22.6 hours in adults.
“Making recombinant VWF available for children marks an important step forward in caring for young patients with VWD,” said Vijay Kumar, MD, acting director of the CBER Office of Therapeutic Products. “This approval is a result of collaborative work between FDA review teams and developers to ensure innovative therapies can reach children alongside adult populations.”
For pediatric hematologists, the recombinant VWF pediatric approval introduces an alternative to plasma-derived treatments, providing greater consistency in pharmacokinetics while avoiding risks associated with plasma-based options. While prophylactic use continues to be restricted to adults, children have been provided with a recombinant therapy for acute and surgical management. This breakthrough should raise standards of care and lower treatment burdens for families and clinicians.
