How it Works

Ivocidenib is an IDH1 inhibitor, which works by blocking a protein called isocitrate dehydrogenase-1 (IDH1),  an enzyme often mutated and overexpressed in some cancers like acute myeloid leukemia (AML). This protein is involved in the production of a molecule called 2-hydroxyglutarate (2-HG), which helps cancer cells grow and spread. By blocking IDH1, ivosidenib helps to slow the growth and spread of AML cells.

How it’s Administered

• Ivocidenib is usually taken as an oral tablet and is often used in combination with other chemotherapy medications. 
• Must not take the tablet with high-fat foods: eggs, butter, dairy products, fried food, or fast food. 

Who Should Take Ivosidenib 

• Patients whose tumor has a certain type of abnormal isocitrate dehydrogenase-1 (IDH1) mutation. This mutation is found in about 10-15% of people with AML and is associated with a poorer prognosis.
• Patients with bile duct cancer that has spread and patients who have already received previous treatment.
• Patients with acute myeloid leukemia that has returned or not improved after previous treatments. 
• 75 years old or older patients diagnosed with AML.
• AML Patients who are not able to have a stem cell transplant.

Who Shouldn’t Take Ivosidenib

• Patients with a severe allergy to ivosidenib or its components.
• Pediatric AML patients because there is not enough evidence about the safety for this population.
• Pregnant women.  It is important to inform your healthcare provider if you are pregnant, or planning to become pregnant before starting treatment with Ivosidenib.
• Ivosidenib may also pass into breast milk, you should suspend breastfeeding when taking this medication. 
• Patients diagnosed with Long QT syndrome (LQTS). A rare inherited heart rhythm disorder that can cause potentially life-threatening irregular heartbeats, known as arrhythmias.

The Most Common Side Effects of Taking Ivosidenib Include:

• Swelling, fatigue, joint pain, difficulty breathing
• Nausea, diarrhea
• Anemia, electrolyte imbalances, high uric acid levels in the blood
• Differentiation syndrome is a very serious side effect that is preventable with proper monitoring and immediate treatment. This syndrome is a reaction between the drug and leukemia, which produces fever, difficulty breathing, weight gain, lung and heart problems. It is generally treated with high-dose steroids. In most cases, the treatment will continue. The syndrome usually occurs during the first month of treatment, with some cases reported following the first dose.

Commonly Used in Acute Myeloid Leukemia With

• Ivosidenib plus azacitidine: Adult patients with an IDH1 mutation and who are not able to have a stem cell transplant or are 75 years old, or older.
• Ivosidenib plus low-dose cytarabine:  Adult patients with an IDH1 mutation and who are not able to have a stem cell transplant or are 75 years old, or older.

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Date last updated: 12/22/22