Ivosidenib targets a specific gene mutation called IDH1, which can affect your bone marrow. IDH1 mutation prevents young blood cells from developing into healthy adult blood cells, which can result in symptoms of acute myeloid leukemia (AML).

Ivosidenib is for use only in adults with an IDH1 mutation. Your doctor will test you for this gene.

Ivosidenib is used to treat AML after other treatments did not work or stopped working.

Ivosidenib is also used to treat AML in adults 75 years and older who have newly-diagnosed AML and cannot use certain chemotherapy treatments because of other health problems.

Ivosidenib is also used to treat bile duct cancer that has progressed or has spread to other parts of the body (metastatic) after treatment with other medicine.

Ivosidenib may also be used for purposes not listed in this medication guide.

Ivosidenib can cause a condition called differentiation syndrome, which affects blood cells and can be fatal if not treated. This condition may occur within 1 days to 3 months after you start taking ivosidenib.

Seek medical help right away if you have symptoms of differentiation syndrome: fever, dizziness, cough, trouble breathing, swelling, rapid weight gain, rash, or decreased urination.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Swallow the tablet whole and do not crush, chew, or break it.

Take the medicine at the same time each day, with or without food.

Do not change your dose or stop taking ivosidenib without your doctor's advice.

Drink plenty of liquids while you are taking ivosidenib.

Do not take ivosidenib with foods that are high in fat, such as butter, bacon, cheese, or whole milk.

If you vomit shortly after taking ivosidenib, do not take another dose. Take your next dose as scheduled.

You may need medical tests to check your heart function.

Store at room temperature away from moisture and heat.

Usual Adult Dose for Acute Myeloid Leukemia:

500 mg orally once a day
Duration of therapy: Until disease progression or unacceptable toxicity

Comments:
-For patients without disease progression or unacceptable toxicity, therapy should continue for at least 6 months to allow time for clinical response.
-Combination regimen: Administration of this drug should start on Cycle 1 Day 1 in combination with azacitidine; the manufacturer product information for azacitidine should be consulted.

Uses:
-In combination with azacitidine or as monotherapy, for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by a US FDA-approved test in patients 75 years and older or who have comorbidities that preclude use of intensive induction chemotherapy
-For the treatment of patients with relapsed or refractory AML with a susceptible IDH1 mutation as detected by a US FDA-approved test

Usual Adult Dose for Cholangiocarcinoma of biliary tract:

500 mg orally once a day
Duration of therapy: Until disease progression or unacceptable toxicity

Use: For the treatment of patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation as detected by a US FDA-approved test

You should not use ivosidenib if you are allergic to it.

Tell your doctor if you have ever had:

• heart disease;

• long QT syndrome (in you or a family member);

• nerve problems;

• an electrolyte imbalance (such as low levels of potassium, sodium, or magnesium in your blood);

• cirrhosis or other liver disease; or

• kidney disease (or if you are on dialysis).

May harm an unborn baby. Use birth control and tell your doctor if you become pregnant.

Ivosidenib can make hormonal birth control less effective, including birth control pills, injections, implants, skin patches, and vaginal rings. Use barrier birth control to prevent pregnancy: condom, diaphragm, cervical cap, or contraceptive sponge.

Pregnancy may be less likely to occur while the mother or the father is using ivosidenib. Women should still use birth control to prevent pregnancy because the medicine can harm an unborn baby.

Do not breastfeed while using ivosidenib, and for at least 1 month after your last dose.

Take the medicine as soon as you can, but skip the missed dose if your next dose is due in less than 12 hours. Do not take two doses within 12 hours.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom).

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. http://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

Unless your doctor tells you otherwise, continue your normal diet.

Make sure you drink plenty of water or other fluids every day while you are taking ivosidenib.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Ivosidenib can cause a condition called differentiation syndrome, which affects blood cells and can be fatal if not treated. This condition may occur within 1 days to 3 months after you start taking ivosidenib.

Seek medical help right away if you have symptoms of differentiation syndrome:

• fever, cough, trouble breathing;

• dizziness;

• rash;

• decreased urination;

• rapid weight gain; or

• swelling in your arms or legs.

Ivosidenib may cause serious side effects. Call your doctor at once if you have:

• fast or pounding heartbeats, fluttering in your chest, shortness of breath, sudden dizziness (like you might pass out);

• jaundice (yellowing of the skin or eyes);

• fluid build-up around the stomach--rapid weight gain, stomach pain and bloating, trouble breathing while lying down;

• low red blood cells (anemia)--pale skin, tiredness, feeling light-headed or short of breath, cold hands and feet;

• high white blood cell counts--fever, weakness, not feeling well, bleeding or bruising, nausea, loss of appetite, weight loss; or

• nervous system problems--numbness, pain, tingling, weakness, burning or prickly feeling, vision or hearing problems, trouble breathing.

Your cancer treatments may be delayed or permanently discontinued if you have certain side effects.

Common side effects of ivosidenib may include:

• irregular heartbeats;

• stomach pain or swelling, nausea, vomiting, loss of appetite;

• diarrhea, constipation;

• fever, tiredness;

• low red blood cell or high white blood cell counts;

• cough, shortness of breath;

• sores in your mouth or throat;

• rash;

• abnormal liver function tests;

• muscle or joint pain; or

• swelling in your arms or legs.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Ivosidenib can cause a serious heart problem. Your risk may be higher if you also use certain other medicines for infections, asthma, heart problems, high blood pressure, depression, mental illness, cancer, malaria, or HIV.

Other drugs may affect ivosidenib, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.

Your doctor will order a lab test before you begin your treatment to see whether your cancer can be treated with ivosidenib.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.