On September 5, 2025, Takeda announced that the U.S. Food and Drug Administration (FDA) approved the supplemental Biologics License Application (sBLA) for VONVENDI® [von Willebrand factor (Recombinant)], expanding its indications to include routine prophylaxis for reducing bleeding episode frequency in adults with von Willebrand Disease (VWD), including Type 1 and 2, and on-demand and perioperative bleeding management in pediatric patients.
VWD, affecting over 3 million individuals in the U.S., is the most common inherited bleeding disorder, caused by deficient or dysfunctional von Willebrand factor (VWF), leading to prolonged bleeding risks from injuries, surgeries, or routine activities. VONVENDI, the only recombinant VWF replacement therapy approved for both adult and pediatric VWD patients, offers a long half-life (22.6 hours in adults, 14.3 hours in children) and demonstrated efficacy in clinical trials, with most non-surgical bleeds resolved with a single infusion.
This approval, supported by three clinical trials (NCT02973087, NCT02932618, NCT03879135) and real-world data, establishes VONVENDI as a critical treatment option for managing bleeding risks and improving quality of life. The expanded indications address unmet needs in VWD management, particularly for prophylaxis in adults with less severe subtypes and pediatric care, marking a significant advancement in treatment standards. Takeda continues to pursue global approvals, including in Japan and the EU, to broaden VONVENDI’s reach.
However, risks such as thromboembolism, hypersensitivity reactions, and neutralizing antibodies require careful monitoring, as outlined in the prescribing information. This milestone underscores Takeda’s commitment to advancing care for the bleeding disorders community, offering hope for better disease management and reduced physical and emotional burdens for patients.
Link: https://www.takeda.com/newsroom/newsreleases/2025/expanded-indication-vonvendi/
