FDA Expands Takeda’s Vonvendi Approval to Broader Range of von Willebrand Disease Patients

FDA Expands Takeda’s Vonvendi Approval to Broader Range of von Willebrand Disease Patients

By, Admin 9 September 2025

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.

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