The World’s Most Common Inherited Bleeding Disorder That Goes Largely Undiagnosed

Imagine a first date or presenting in front of a large group and suddenly developing a serious, uncontrollable nosebleed that you don’t know how to stop. Or having heavy menstrual periods that last for weeks, even months.

You don’t have to imagine those kinds of situations if you have von Willebrand disease (VWD). That may be your reality.

Most people have never even heard of VWD, the world’s most common inherited bleeding disorder. The condition, named after the Finnish physician who identified it in the 1920s, affects an estimated 1 in 100 people globally, impacting men and women equally.

People with VWD have a problem with a key element in the blood-clotting process – von Willebrand factor (VWF), which binds other proteins together to help create a successful clot and stop a bleed. They may not make enough VWF or it may not work normally. Because of this, the disease primarily affects the body’s soft tissues, and VWD patients can suffer complications including frequent, severe nose bleeds, extremely heavy menstrual periods and bleeding gums.

No one simple blood test exists to identify people with the disease, and many physicians have little or no familiarity with the condition because symptoms can be very mild. It can be difficult to diagnose because bleeds manifest differently among people with VWD and are often misdiagnosed before VWD is considered. Furthermore, levels of VWF in the body vary over time and can cause different outcomes of tests. Many patients can go years without knowing they even have VWD.

Once a diagnosis is received, treatment depends on diagnosis and severity. Available treatment options for individuals with VWD include vasopressin analogues, antifibrinolytics, hormone therapy, and plasma-derived VWF/Factor VIII (FVIII) replacement treatments that contain FVIII.

Recently introduced VONVENDI [von Willebrand factor (Recombinant)] is the only treatment available that features recombinant von Willebrand factor – a genetically engineered protein that replaces a patient’s natural factor. This allows healthcare providers to dose recombinant von Willebrand factor (rVWF) with or without recombinant Factor VIII (rFVIII) based on each patient’s individual needs.

“VONVENDI dosing is designed to meet the body’s individual requirements, offering patients and healthcare providers a new option to help manage this challenging chronic disease,” says Ralph Gruppo, M.D., Director, Comprehensive Hemophilia and Thrombosis Center at the Cincinnati Children’s Hospital.

In a Phase III trial, VONVENDI demonstrated 100 percent treatment success (95% CI 81.5 to 100; n=18), which was defined with the use of a ratings scale for the treatment of bleeds. The scale measured efficacy as either “Excellent,” “Good,” “Moderate,” or “None.” Its ability to control bleeding was rated as “Excellent” in 96.9% of episodes and “Good” in 3.1% of episodes. The most common adverse reaction observed in greater than two percent of patients in clinical trials was generalized pruritus.

The launch of VONVENDI offers a treatment option for adults living with VWD to treat and control bleeding episodes. Learn more about VWD and VONVENDI at vonvendi.com.

VONVENDI [Von Willebrand Factor (Recombinant)] Important Information

Indication
VONVENDI is used to treat and control bleeding episodes in adults (age 18 years and older) diagnosed with von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

You should not use VONVENDI if you:

• Are allergic to any ingredients in VONVENDI.
• Are allergic to mice or hamsters.

Tell your healthcare provider if you are pregnant or breastfeeding because VONVENDI may not be right for you.

Your first dose of VONVENDI for each bleeding episode may be administered with a recombinant factor VIII as instructed by your healthcare provider.

You should tell your healthcare provider if you:

• Have or have had any medical problems.
• Take any medicines, including prescription and nonprescription medicines, such as over-the-counter medicines, supplements or herbal remedies.
• Have any allergies, including allergies to mice or hamsters.
• Are breastfeeding. It is not known if VONVENDI passes into your milk and if it can harm your baby.
• Are pregnant or planning to become pregnant. It is not known if VONVENDI can harm your unborn baby.
• Have been told that you have inhibitors to von Willebrand factor (because VONVENDI may not work for you).
• Have been told that you have inhibitors to blood coagulation factor VIII.

Your body can form inhibitors to von Willebrand factor or factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop VONVENDI or FVIII from working properly. Consult with your healthcare provider to make sure you are carefully
monitored with blood tests for the development of inhibitors to von Willebrand factor or factor VIII.

You can have an allergic reaction to VONVENDI. Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting.

Side effects that have been reported with VONVENDI include: nausea, tingling or burning at infusion site, chest discomfort, dizziness, hot flashes, itching, nausea, high blood pressure, muscle twitching, unusual taste, and increased heart rate.

Tell your healthcare provider about any side effects that bother you or do not go away.

You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

For full Prescribing Information for VONVENDI visit:
http://baxalta.com/assets/documents/VONVENDI_PI.pdf.