FDA Approves Vonjo™ (pacritinib) for Intermediate- or High-Risk Myelofibrosis With Severe Thrombocytopenia

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Vonjo™ (pacritinib) for certain people with intermediate- or high-risk myelofibrosis. Myelofibrosis is a rare type of leukemia. It is a blood cancer that impacts the bone marrow, or the material inside our bones.^1,2

Vonjo is approved for adults who have myelofibrosis with severe thrombocytopenia. This means they have a very low blood platelet count. To receive Vonjo, their platelet count must be below 50 x 109/L.³

Vonjo may be a good treatment option for people with myelofibrosis and low platelet counts. This is important because low platelet counts can cause complications and challenges. These include:^3,4

• Higher risk of bleeding
• Worse symptoms
• More advanced disease and higher risk of complications
• Less treatment options

Accelerated approval is granted to new drugs that are very promising and could make a positive impact on the treatment of serious conditions. There have been limited options for people with intermediate- or high-risk myelofibrosis with severe thrombocytopenia. Vonjo may be a new treatment option to help these people.¹

What are the ingredients in Vonjo?

The active ingredient in Vonjo is pacritinib.³

How does Vonjo work?

Vonjo is a type of drug called a kinase inhibitor. Kinase inhibitors are often used to treat different types of cancers. A kinase is a type of protein that cells use for many different functions.^3,5

Vonjo works by targeting certain types of kinases that are more present in cancer cells. Targeting these kinases makes it harder for these cells to grow. This helps reduce the spread and impact of cancers in our bodies.^3,5

Vonjo is an oral kinase inhibitor. This means it is taken as a pill.^3,5

Evidence for Vonjo

Vonjo was approved based on results from a clinical trial. The trial studied the symptoms and spleen size of the people enrolled in the trial. Spleen size was studied because myelofibrosis can cause an enlarged spleen. This means that a smaller spleen was a sign that the treatment was working.⁴

The trial found that about 30 percent of the people in the trial taking Vonjo had smaller spleens after treatment with the drug. Also, about 10 percent more of those who took Vonjo had improved symptoms than those who took other treatments.⁴

What are the possible side effects of Vonjo?

The most common side effects of Vonjo include:³

• Nausea and vomiting
• Low red blood cell count (anemia)
• Swelling in the legs, hands, and feet

Vonjo may also cause serious side effects that can be life threatening, including:³

• Severe bleeding
• Severe diarrhea
• Irregular heartbeat
• Low blood platelet levels
• Serious heart events such as stroke or heart attack
• Blood clots
• Increased risk for other cancers
• Infections

Stop Vonjo and contact your doctor right away if you have any signs of serious side effects, including:³

• Bleeding
• Feelings of dizziness or lightheadedness
• Symptoms of a heart attack or stroke
• Symptoms of blood clots
• Symptoms of an infection

These are not all the possible side effects of Vonjo. Talk to your doctor about what to expect when taking Vonjo. You should also call your doctor if you have any changres that concern you when taking Vonjo.

Things to know about pacritinib

Your doctor will do blood tests to check your blood counts before and during treatment with Vonjo.³

Before starting Vonjo, tell your doctor if you have ever had blood clogs in the veins of your legs or lungs.³

Vonjo may affect fertility in men and make it difficult to father a child. Talk to your doctor if this is a concern for you.³

Before beginning treatment for myelofibrosis, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

For more information, read the full prescribing information of Pacritinib.