ICYMI: VONVENDI Approved for Von Willebrand Disease

There are three subsets of Von Willebrand disease (vWD), a genetic bleeding disorder. Type 3 is considered to be the most severe form of vWD, culminating in intense or severe bleeding episodes. In late January 2022, biopharmaceutical company Takeda Pharmaceutical Company Limited (“Takeda”) shared that its treatment VONVENDI was now FDA-approved as a prophylaxis for the treatment of vWD.


VONVENDI is the first approved recombinant von Willebrand factor (rVWF) replacement therapy to be used for routine prophylaxis. The treatment, administered via infusion, is designed to lower the amount of bleeding episodes experienced by adult patients. Additionally, VONVENDI can be used on-demand or during the perioperative process to reduce the risk of severe bleeds.

The FDA approval hinged on data from a study which evaluated VONVENDI for Type 3 von Willebrand disease. Altogether, 10 adults enrolled in the study. Ultimately, the research highlighted how VONVENDI significantly reduced severe bleeding events.

While the therapy is relatively safe and well-tolerated, some adverse reactions may occur. These include:

  • High blood pressure
  • Nausea and vomiting
  • Blood clots
  • Headaches
  • Rash or hives
  • Infusion site reaction
  • Chest pain or tightness (which may come with difficulty breathing)
  • Joint pain

Learn more about VONVENDI.

Von Willebrand Disease (vWD)

There are over 300 VWF gene mutations associated with von Willebrand disease (vWD), an inherited bleeding disorder which affects the blood’s clotting ability. First described in 1925, von Willebrand disease is considered to be the most common inherited bleeding disorder. This condition affects males and females equally. Those with vWD do not have enough von Willebrand factor (VWF), a protein which helps hold platelets together and carry factor VIII through the bloodstream.

There are three subsets of von Willebrand disease. An estimated 75% of those with vWD have Type 1, which is the most mild form. This form is characterized by lower than average VWF levels. Next, those with Type 2 have normal VWF levels – but the protein does not function properly. Type 2 is also associated with various subtypes, include 2A, 2B, and 2N. Finally, there is Type 3 VWD, the most rare and severe form. In this form, there is little to no VWF protein in the blood.

Due to the fact that a majority of people have mild symptoms, many believe that vWD is highly under-diagnosed. In fact, an estimated 90% of those with this condition have not received a diagnosis. Symptoms include:

  • Easy bruising and bleeding
  • Bleeding from gums
  • Bloody stool or urine
  • Long or heavy menstruation
  • Frequent, difficult-to-stop nosebleeds
  • Excessive bleeding following an injury, surgery, or dental procedure
  • Joint and muscle bleeds
    • Note: These bleeds may culminate in limited mobility, inflammation and heat, and pain.
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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