FDA approves first recombinant VWF for pediatric von Willebrand disease

The FDA has expanded the indication for recombinant von Willebrand factor (VWF), marketed as Vonvendi, to now include children with von Willebrand disease (VWD) for both the treatment of acute bleeding episodes and use in surgical procedures.

Announced on September 5, 2025, the decision establishes recombinant VWF as the first non–plasma-derived therapy available for pediatric patients with VWD in the United States. This significantly broadens its reach beyond prior approvals, which were restricted to adults—initially for on-demand treatment of bleeding, later for perioperative use, and most recently for routine prophylaxis in adults 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 (CBER). “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.”

Before the September 2025 update, recombinant VWF had received 3 separate FDA approvals over the past decade. Its original authorization came in December 2015, when it was cleared for treating bleeding episodes in adults with VWD based on findings from 2 trials that enrolled 69 patients. In 2018, the approval expanded to cover perioperative bleeding management in adults following a multicenter, phase 3 study that evaluated its use with or without recombinant FVIII during elective procedures. A further milestone came in 2022, when prophylactic use was approved for adults with severe type 3 VWD receiving on-demand therapy.

According to Takeda Pharmaceuticals, the pediatric approval is backed by findings from 3 clinical studies, including a phase 3 trial specifically enrolling children and a phase 3b continuation study that included both children and adults. Real-world evidence was also used to support the expanded indication. Trial results showed that most nonsurgical bleeds in pediatric patients were effectively managed with a single infusion, with a reported half-life of 14.3 hours in children 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, this approval introduces a recombinant alternative to plasma-derived VWF products, offering more consistent pharmacokinetics and eliminating risks inherent to plasma-based therapies. Although prophylaxis remains an option only for adults at this time, children with VWD now have access to a recombinant therapy for acute management and surgical settings—an advance expected to improve care and ease treatment burdens for patients and families alike.

References

1. FDA approves expanded use of Vonvendi for von Willebrand disease, including for certain uses for children. News release. FDA. September 5, 2025. Accessed September 5, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-expanded-use-vonvendi-von-willebrand-disease-including-certain-uses-children

2. U.S. FDA approves expanded indication for VONVENDI [von Willebrand factor (recombinant)] for adults and children with von Willebrand disease. News release. Takeda. September 5, 2025. Accessed September 5, 2025. https://www.takeda.com/newsroom/newsreleases/2025/expanded-indication-vonvendi