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news.Takeda Pharmaceuticals has received the US Food and Drug Administration (FDA) approval for the supplemental Biologics License Application (sBLA) of an expanded indication for Vonvendi, for routine prophylaxis in adults with von Willebrand disease (VWD), including Types 1 and 2.
In trials, most non-surgical bleeds in adults and children were controlled with one infusion. Credit: National Cancer Institute on Unsplash.
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VWD is the most prevalent inherited bleeding disorder in the US, affecting over three million individuals, with both men and women equally susceptible.
Vonvendi is also approved for on-demand and perioperative management of bleeding in children with VWD.
Previously, it was indicated solely for on-demand and perioperative use in adult people with VWD and for routine prophylaxis in those with severe Type 3 VWD receiving on-demand therapy.
It remains the only recombinant VWF replacement therapy with approved indications for both adults and children with VWD.
Vonvendi is administered via infusion to replace missing or defective VWF. Clinical data indicate a half-life of 22.6 hours in adults and 14.3 hours in children.
In clinical trials, most non-surgical bleeds in adults and children were controlled with just one infusion.
Vonvendi prophylaxis study investigator Jonathan Roberts said: “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.
“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.”
The FDA’s decision is informed by data from three trials: a Phase III study in adults, a Phase III study in children, and a Phase IIIb continuation trial, supplemented by supporting real-world evidence.
Takeda has additionally submitted applications to extend the use of Vonvendi for paediatric on-demand and perioperative management in Japan, as well as to register Veyvondi (vonicog alfa) for paediatric on-demand use within the European Union.
