Written By: Pharmacally Medical News Desk
On September 5, 2025, the U.S. Food and Drug Administration (FDA) approved a broader set of uses for Vonvendi (recombinant von Willebrand factor), including the first pediatric indication in the U.S.
The expanded approval allows Vonvendi to be used more broadly across patient groups. For adults aged 18 years and older, it is now cleared for routine prophylaxis, meaning preventative infusions to reduce bleeding episodes across all types of von Willebrand disease, not just Type 3. For children, the FDA has authorized its use for on-demand treatment of bleeding episodes as well as for perioperative use around surgical procedures. Importantly, this marks the first time a recombinant, non-plasma-derived von Willebrand factor has been approved for pediatric patients in the United States.
This approval matters for several reasons. First, it expands access by making Vonvendi the only recombinant von Willebrand factor product approved for both adults and children in the United States. It also simplifies treatment, as trial data showed that most non-surgical bleeds required just one infusion, supported by the drug’s long half-life of about 22.6 hours in adults and 14.3 hours in children. Finally, the decision reflects the FDA’s adaptive approach to rare diseases. As FDA director Vinay Prasad, M.D., M.P.H., explained, the agency acts promptly when there is a strong scientific basis combining a plausible mechanism of action, robust pharmacology, and supportive clinical data even when the evidence comes from smaller studies.
The FDA’s decision was based on evidence from three Phase 3 clinical trials and supporting real-world data. In adults, a pivotal trial (NCT02973087) demonstrated the effectiveness of Vonvendi for prophylaxis by significantly reducing bleeding episodes. A separate pediatric study (NCT02932618) confirmed its safety and efficacy for on-demand treatment and surgical use in children. These findings were reinforced by a continuation study (NCT03879135) that included both adults and children, providing consistent results across age groups. Together, these studies showed reliable control of bleeding, a favorable safety profile, and clear benefit over existing options, forming the clinical basis for the expanded approval.
The safety profile of Vonvendi was consistent with expectations and generally well tolerated across studies. The most common side effects, reported in at least 2% of participants, included headache, nausea, vomiting, dizziness, and itchy skin (pruritus). No unexpected safety concerns were identified, and overall, the treatment was considered safe for both adult and pediatric patients with von Willebrand disease.
According to the FDA’s approval letter, Takeda must update Vonvendi’s labeling, including the package insert and instructions for use, and submit these materials in the required structured product labeling format within 14 days.
This approval opens important new options for people living with von Willebrand disease. Adults across all disease types can now benefit from routine preventative treatment, while children finally have access to the first recombinant therapy designed for their condition. The drug’s long half-life means fewer infusions, offering added convenience and consistency compared to plasma-derived alternatives. Just as significant, the FDA’s decision reflects a flexible, science-driven approach to rare diseases, showing that strong biologic rationale and supportive data even from smaller studies can bring meaningful treatments to patients sooner. Together, these changes mark a step forward in both patient care and regulatory innovation.
Reference
FDA Approves Expanded Use of Vonvendi for von Willebrand Disease, Including for Certain Uses for Children, 05 Sept 2025, USFDA, https://www.fda.gov/news-events/press-announcements/fda-approves-expanded-use-vonvendi-von-willebrand-disease-including-certain-uses-children