Takeda announced that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Vonvendi [von Willebrand factor (Recombinant)], expanding its indications.
Expanded Indications
Vonvendi is now approved for:
- Adults with VWD (Type 1, 2, and severe Type 3) → routine prophylaxis to reduce the frequency of bleeding episodes.
- Paediatric patients with VWD → on-demand treatment and perioperative management of bleeding.
This makes Vonvendi the only recombinant von Willebrand Factor (VWF) therapy approved for both adults and children.
Background on Vonvendi
- Previously approved for:
- On-demand and perioperative use in adults with VWD.
- Routine prophylaxis in severe Type 3 adult patients. - Unique features:
- Infused replacement therapy for missing or dysfunctional VWF.
- Long half-life: 22.6 hours in adults, 14.3 hours in children. - Clinical data: Most bleeds in trials were treated with a single infusion, adults (157/192) and children (80/104).
Clinical Significance
VWD (von Willebrand Disease):
- The most common inherited bleeding disorder, affecting >3 million people in the US.
- Caused by low or dysfunctional VWF.
- Leads to life-threatening bleeds, heavy menstrual bleeding, prolonged nosebleeds, GI bleeds, complications after surgery, and childbirth risks.
Jonathan C. Roberts, MD, Bleeding and Clotting Disorders Institute:
“Prophylaxis as the standard of care for all adults with VWD is a significant step forward, reducing spontaneous and joint bleeds and easing daily burdens.”
Data Supporting Approval
FDA’s decision was based on results from:
- Phase 3 trial in adults with VWD (NCT02973087)
- Phase 3 trial in children with VWD (NCT02932618)
- Phase 3b continuation study in adults & children (NCT03879135)
- Supported by real-world evidence.
Corporate Commentary
Cheryl Schwartz, SVP, Takeda US Rare Disease Business Unit:
“Vonvendi’s expanded use will help more patients — both adults and children — manage bleeding risk, reinforcing Takeda’s long-standing commitment to the blood and bleeding disorders community.”
Next Steps
- Takeda has filed for approvals in Japan (paediatric on-demand & perioperative use) and in the EU (Veyvondi for paediatric on-demand use).
About Takeda
Takeda is an independent, global pharmaceutical company focused on rare diseases, oncology, neuroscience, vaccines, plasma-derived therapies, and GI & inflammation. The company’s purpose is to deliver life-transforming treatments while advancing global health and sustainability.
