Vonvendi: A new kind of von Willebrand disease treatment

Vonvendi is the first synthetic treatment for preventing bleeding episodes and managing surgical bleeding in adults and children with von Willebrand disease (VWD). Learn who it’s prescribed for, how it works, and what the latest research shows regarding its safety and effectiveness.

Von Willebrand disease (VWD) is the most common inherited bleeding disorder. Vonvendi is the first recombinant (human-made) version of a protein called von Willebrand factor, which is necessary for blood clotting. It’s made in a lab without human plasma or blood. It provides a new option to plasma-based products, one that eliminates the risk of spreading blood-borne viruses.

Vonvendi comes as a powder in single-dose vials that a healthcare professional will mix with liquid to make a solution for intravenous (IV) infusion. (This is an injection into a vein given over a period of time.) Vonvendi infusions can be given in a hospital or clinic, or you can be trained to give yourself infusions at home.

Vonvendi dosing is highly personalized and calculated by your doctor based on several factors. These include your body weight in kilograms (kg) and the type of bleed you’re receiving Vonvendi to treat.

In some instances (especially for your first dose for a bleeding episode), your doctor will prescribe Vonvendi together with another clotting factor called Factor VIII.

Vonvendi is typically well tolerated, but side effects can occur.

The most common side effects (reported by 2% or more of people participating in the drug’s clinical trials) include headache, vomiting, nausea, dizziness, and widespread itchiness.

Some people also experience infusion reactions while receiving doses of Vonvendi. These may include: burning or tingling at the injection site, hot flashes, chest discomfort, or rapid heart rate.

Seek emergency medical care if you experience symptoms of a severe allergic reaction (anaphylaxis) such as swelling of the face and throat, chest tightness, wheezing, or hives.

Vonvendi has warnings regarding the risks of allergic reaction, blood clots, and neutralizing antibodies.

You should not use this medication if you’re allergic to it or any of its ingredients. Because of how Vonvendi is made, people with an allergy to hamster or mouse proteins should avoid this medication or be extremely cautious when taking it.

Because this medication helps blood to clot, there’s a chance it could work too well and lead to clots in your lungs or veins, or cause a stroke or heart attack. This risk is higher in people who have other risk factors and those who are receiving very high Vonvendi doses.

It’s possible that your immune system could mistake this medication as a threat and create antibodies to neutralize it. Your doctor will test you for a high level of these inhibitors if Vonvendi’s effectiveness in treating your condition begins to decline.

Vonvendi isn’t known to interact with any other drugs. However, this medication helps to increase your body’s Factor VIII levels. So if you take an extra Factor VIII treatment along with Vonvendi, your blood levels of this protein could become too high, increasing your risk of blood clots.

There are a few other important things to know about this medication.

It isn’t currently known whether Vonvendi can harm a fetus or if it passes into breast milk. If you’re pregnant, planning a pregnancy, or nursing, discuss the risks and benefits of this treatment with your doctor.

Vonvendi powder should be stored in a refrigerator at 36°F to 46°F (2°C to 8°C), or at room temperature no higher than 86°F (30°C).

Note: Once you mix the powder with the liquid, you must inject the solution within 3 hours.

Because Vonvendi is a specialty drug made using genetically engineered cells in a highly controlled environment, the cost is significantly higher than with mass-produced retail medications.

There is also a non-profit, third-party assistance program for people with government insurance (such as Medicare and Medicaid), who aren’t eligible for the manufacturer’s co-pay assistance. This Von Willebrand disease fund is administered by the PAN Foundation and Patient Advocate Foundation. If eligible, you could receive an initial grant of $8,800 per year (which can often be renewed for up to $17,600) to help cover Vonvendi co-pays.

Vonvendi is distributed by specialty pharmacies such as Accredo and CVS Specialty, who have teams dedicated to getting this type of medication approved and funded.

Talk with your doctor or specialty pharmacist if you have questions about payment assistance for Vonvendi treatment.

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