Takeda has received the US Food and Drug Administration’s (FDA) approval for expanding the indication of Vonvendi to include children with von Willebrand Disease (VWD).
Previously, Vonvendi was limited to on-demand treatment and perioperative use in adults and routine prevention in those with severe type-three VWD.
The current approval expands the indication to include on-demand and perioperative bleeding management in paediatric patients, along with routine prophylaxis for adults with type-one and type-two VWD.
With the expanded approval, Vonvendi becomes the only recombinant von Willebrand Factor replacement therapy approved for both adults and children.
The US health regulator approved Vonvendi under its Priority Review process, following the orphan drug designation.
Takeda US rare disease business unit lead and US commercial operations senior vice president Cheryl Schwartz said: “Children and adults living with VWD face unique challenges, including bleeds that can disrupt daily activities like school, work or spending time with loved ones, often leading to physical and emotional burdens.
“We are proud to have supported adults with VWD since VONVENDI’s initial approval in 2015 and will now be able to help even more patients manage their condition.
“This milestone highlights Takeda’s unwavering commitment to the blood and bleeding disorders community as we strive to deliver innovative therapies.”
VWD is a common inherited bleeding disorder that results in low or dysfunctional von Willebrand factor, leading to potentially severe bleeding complications.
The FDA’s decision is supported by data from three clinical trials, including Phase 3 studies in both adults and children with VWD, and a Phase 3b continuation trial.
In the clinical trials, most non-surgical bleeds required only one infusion of Vonvendi, with notable effectiveness in both adult and pediatric groups.
The most frequent side effects reported in more than 2% of patients include headaches, vomiting, nausea, dizziness, and generalised pruritus.
Furthermore, Takeda is also pursuing approvals in Japan for paediatric bleeding management and in the European Union (EU) for on-demand use of Veyvondi.
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.”
