Takeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for VONVENDI^® [von Willebrand factor (Recombinant)], expanding the indication to include routine prophylaxis to reduce the frequency of bleeding episodes in adults with von Willebrand Disease (VWD), including those with Type 1 and 2 disease, and on-demand and perioperative management of bleeding in pediatric patients with VWD. VONVENDI was previously approved for on-demand and perioperative use in adults with VWD and routine prophylactic use in adults with severe Type 3 VWD receiving on-demand therapy.

VONVENDI is now available for use in a broad range of VWD patients and is the only recombinant von Willebrand Factor (VWF) replacement therapy with approved indications in both adults and children with VWD.

VWD is the most common bleeding disorder impacting more than 3 million people in the U.S. alone. It occurs equally among men and women. People with VWD have low levels of or dysfunctional von Willebrand factor (VWF), a protein that helps blood clot. This can lead to life-threatening bleeding – either from an injury or after surgery, gastrointestinal bleeding, dental procedures or childbirth – prolonged nosebleeds, heavy or long menstrual bleeding and easy bruising, which can have a significant impact on quality of life.

VONVENDI is an infused medicine designed to replace the body's missing or dysfunctional VWF. VONVENDI has a uniquely long half-life (22.6 hours [50 IU/kg] for adults and 14.3 hours for children). In clinical trials, the majority of non-surgical bleeds were treated with one VONVENDI infusion in both adults (157/192) and children (80/104).

“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,” said Jonathan C. Roberts, M.D., associate medical and research director at the Bleeding and Clotting Disorders Institute, associate professor of pediatrics and medicine at the University of Illinois College of Medicine at Peoria, and an investigator in the VONVENDI prophylaxis study. “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.”

This approval is based on data from three clinical trials – a Phase 3 trial in adults with VWD (NCT02973087), a Phase 3 study in children with VWD (NCT02932618) and a Phase 3b continuation trial in adults and children with VWD (NCT03879135) – as well as supportive real world data.¹ Please see the VONVENDI full Prescribing Information for details on the established clinical profile.

“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,” said Cheryl Schwartz, Senior Vice President, U.S. Rare Disease Business Unit Lead and U.S. Commercial Operations at Takeda. “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.”

Takeda submitted applications seeking approval to expand the indications of VONVENDI for on-demand and perioperative management of bleeding in pediatric patients with VWD in Japan and VEYVONDI™ (vonicog alfa) for on-demand use in pediatric patients with VWD in the European Union.