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WHAT IS IDH1?

Learn about IDH1 mutations and how they can affect your cancer

In healthy cells, the IDH1 gene makes the IDH1 protein, which plays a role in normal chemical reactions within cells. When the IDH1 gene changes due to a genetic mutation, the IDH1 protein functions differently.

This mutated IDH1 protein may promote the development of certain types of cancers, including acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and cholangiocarcinoma (CCA).

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Targeted Therapy

TIBSOVO is the first treatment that targets IDH1 mutations in AML, MDS, and CCA

TIBSOVO specifically targets the mutated IDH1 protein. Unlike chemotherapy, which kills normal and healthy cells, TIBSOVO only targets cells with mutated IDH1.

IDH1 mutations can be found by doing biomarker testing, which involves your doctor sending a sample of your blood (for AML and MDS testing) or a piece of your tumor (for CCA testing) to a laboratory.

TIBSOVO has been proven to help people with AML, MDS, and CCA who have IDH1 mutations.

Talk to your doctor about biomarker testing and find out if TIBSOVO is right for you

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View TIBSOVO results in MDS
References: 1. National Cancer Institute. NCI Dictionary of Cancer Terms: IDH1 gene. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/idh1-gene. Accessed July 16, 2026. 2. Mondesir J, Willekens C, Touat M, de Botton S. IDH1 and IDH2 mutations as novel therapeutic targets: current perspectives. J Blood Med. 2016;7:171-180. doi:10.2147/JBM.S70716 3. Yang H, Ye D, Guan KL, Xiong Y. IDH1 and IDH2 mutations in tumorigenesis: mechanistic insights and clinical perspectives. Clin Cancer Res. 2012;18(20):5562-5571. doi:10.1158/1078-0432.CCR-12-1773 4. Tibsovo. Package insert. Servier Pharmaceuticals LLC; 2023. 5. Popovici-Muller J, Lemieux RM, Artin E, et al. Discovery of AG-120 (ivosidenib): a first-in-class mutant IDH1 inhibitor for the treatment of IDH1 mutant cancers. ACS Med Chem Lett. 2018;9(4):300-305. doi:10.1021/acsmedchemlett.7b00421 6. American Cancer Society. Chemotherapy. https://www.cancer.org/cancer/managing-cancer/treatment-types/chemotherapy.html. Accessed July 16, 2026. 7. DiNardo CD, Ravandi F, Agresta S, et al. Characteristics, clinical outcome, and prognostic significance of IDH mutations in AML. Am J Hematol. 2015;90(8):732-736. doi:10.1002/ajh.24072 8. Thol F, Weissinger EM, Krauter J, et al. IDH1 mutations in patients with myelodysplastic syndromes are associated with an unfavorable prognosis. Haematologica. 2010;95(10):1668-1674. doi:10.3324/haematol.2010.025494 9. Salati M, Caputo F, Baldessari C, et al. IDH signalling pathway in cholangiocarcinoma: from biological rationale to therapeutic targeting. Cancers (Basel). 2020;12(11):3310. doi:10.3390/cancers12113310 10. de Haas V, Ismaila N, Advani A, et al. Initial diagnostic work-up of acute leukemia: ASCO clinical practice guideline endorsement of the College of American Pathologists and American Society of Hematology guideline. J Clin Oncol. 2019;37(3):239-253. doi:10.1200/JCO.18.01468 11. US Food and Drug Administration. List of cleared or approved companion diagnostic devices (in vitro and imaging tools). https://www.fda.gov/medical-devices/in-vitro-diagnostics/list-cleared-or-approved-companion-diagnostic-devices-in-vitro-and-imaging-tools. Accessed July 21, 2026. 12. US Food and Drug Administration. Oncomine Dx Target Test – P160045/S031. https://www.fda.gov/medical-devices/recently-approved-devices/oncomine-dx-target-test-p160045s031. Accessed July 21, 2026. 13. National Cancer Institute. NCI Dictionary of Cancer Terms: biomarker testing. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/biomarker-testing. Accessed July 16, 2026.
 

What is TIBSOVO?

TIBSOVO is a prescription medicine used to treat adults with an isocitrate dehydrogenase-1 (IDH1) mutation with:

  • acute myeloid leukemia (AML):
    • with newly diagnosed AML treated in combination with azacitidine or alone who are 75 years or older or who have health problems that prevent the use of certain chemotherapy treatments
    • when the disease has come back or has not improved after previous treatment(s)
  • myelodysplastic syndromes (MDS):
    • when the disease has come back or has not improved after previous treatment(s)
  • bile duct cancer (cholangiocarcinoma):
    • with bile duct cancer that has spread and
    • who have already received previous treatment(s)

Your healthcare provider will perform a test to make sure that TIBSOVO is right for you. It is not known if TIBSOVO is safe and effective in children.

 

IMPORTANT SAFETY INFORMATION

TIBSOVO may cause serious side effects, including:

  • Differentiation syndrome. Differentiation syndrome is a common condition that affects your blood cells and may be life-threatening or lead to death. Differentiation syndrome in adults with AML and MDS has happened as early as 1 day and up to 3 months after starting TIBSOVO. Call your healthcare provider or go to the nearest hospital emergency room right away if you develop any of the following symptoms of differentiation syndrome during treatment with TIBSOVO:
  • fever
  • cough
  • trouble breathing
  • rash
  • decreased urination
  • dizziness or lightheadedness
  • rapid weight gain
  • swelling of your arms and legs

If you develop signs and symptoms of differentiation syndrome, your healthcare provider may treat you with a corticosteroid medicine or a medicine called hydroxyurea and may monitor you in the hospital.

  • Changes in the electrical activity of your heart called QTc prolongation. QTc prolongation can cause irregular heartbeats that can be life-threatening. Your healthcare provider will check the electrical activity of your heart with a test called an electrocardiogram (ECG) before and during treatment with TIBSOVO. Tell your healthcare provider right away if you feel dizzy, lightheaded, or faint
  • Guillain-Barré syndrome has happened in people treated with TIBSOVO. Your healthcare provider will monitor you for nervous system problems and will permanently stop your treatment with TIBSOVO if you develop Guillain-Barré syndrome. Tell your healthcare provider right away if you develop any signs or symptoms of Guillain-Barré syndrome, including:
  • weakness or tingling feeling in your legs, arms, or upper body
  • numbness and pain on one side or both sides of your body
  • any changes in your ability to see, touch, hear, or taste
  • burning or prickling sensation
  • difficulty breathing

The most common side effects of TIBSOVO when used in combination with azacitidine or alone in adults with AML include:

  • changes in certain blood cell counts
  • diarrhea
  • increased blood sugar
  • fatigue
  • changes in certain liver function tests
  • swelling of arms or legs
  • decreased levels of electrolytes in the blood
  • nausea
  • vomiting
  • decreased appetite
  • joint pain
  • shortness of breath
  • uric acid increased
  • stomach (abdominal) pain
  • changes in certain kidney function tests
  • pain or sores in your mouth or throat
  • rash
  • irregular heart rhythm or heartbeat (QTc prolongation)
  • differentiation syndrome
  • muscle pain

The most common side effects of TIBSOVO when used in adults with MDS include:

  • changes in certain kidney function tests
  • changes in certain blood cell counts
  • joint pain, back pain, or neck pain
  • decreased levels of albumin in the blood
  • changes in liver function tests
  • fatigue
  • diarrhea
  • cough
  • decreased levels of sodium in the blood
  • pain or sores in your mouth or throat
  • decreased appetite
  • muscle pain
  • decreased levels of phosphorus in the blood
  • itchy skin
  • rash

The most common side effects of TIBSOVO in adults with cholangiocarcinoma include:

  • fatigue
  • nausea
  • abdominal pain
  • diarrhea
  • cough
  • decreased appetite
  • fluid and swelling in your stomach area
  • vomiting
  • hemoglobin decreased (anemia)
  • rash
  • changes in liver function tests

Your healthcare provider will do blood tests before you start and during treatment with TIBSOVO. Your healthcare provider may decrease, temporarily hold, or permanently stop your treatment with TIBSOVO if you develop certain side effects.

TIBSOVO may cause fertility problems in females and males, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility.

These are not all of the possible side effects of TIBSOVO. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before taking TIBSOVO, tell your healthcare provider about all of your medical conditions, including if you:

  • have any heart problems, including a condition called long QT syndrome
  • have problems with abnormal electrolytes, such as sodium, potassium, calcium, or magnesium levels
  • have nervous system problems
  • have problems with your kidneys or are on dialysis
  • have any liver disorders, including cirrhosis
  • are pregnant or plan to become pregnant. TIBSOVO may cause harm to your unborn baby. You should avoid becoming pregnant during treatment with TIBSOVO. Tell your healthcare provider right away if you become pregnant or think you might be pregnant during treatment with TIBSOVO
  • are breastfeeding or plan to breastfeed. It is not known if TIBSOVO passes into your breast milk. Do not breastfeed during your treatment with TIBSOVO and for 1 month after your last dose of TIBSOVO

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Especially tell your healthcare provider if you take hormonal contraceptives. TIBSOVO may affect how hormonal contraceptives work and may cause them to not work as well.

Please see Full Prescribing Information, including Boxed WARNING for AML and MDS patients and Medication Guide.

Last update: September 2026

Information on this website is reviewed through established medical, legal, and regulatory processes to support accurate, balanced, and label-consistent communication about TIBSOVO.

 

What is TIBSOVO?

TIBSOVO is a prescription medicine used to treat adults with an isocitrate dehydrogenase-1 (IDH1) mutation with:

  • acute myeloid leukemia (AML):
    • with newly diagnosed AML treated in combination with azacitidine or alone who are 75 years or older or who have health problems that prevent the use of certain chemotherapy treatments
    • when the disease has come back or has not improved after previous treatment(s)
  • myelodysplastic syndromes (MDS):
    • when the disease has come back or has not improved after previous treatment(s)
  • bile duct cancer (cholangiocarcinoma):
    • with bile duct cancer that has spread and
    • who have already received previous treatment(s)

Your healthcare provider will perform a test to make sure that TIBSOVO is right for you. It is not known if TIBSOVO is safe and effective in children.

IMPORTANT SAFETY INFORMATION

TIBSOVO may cause serious side effects, including:

  • Differentiation syndrome. Differentiation syndrome is a common condition that affects your blood cells and may be life-threatening or lead to death. Differentiation syndrome in adults with AML and MDS has happened as early as 1 day and up to 3 months after starting TIBSOVO. Call your healthcare provider or go to the nearest hospital emergency room right away if you develop any of the following symptoms of differentiation syndrome during treatment with TIBSOVO:

IMPORTANT SAFETY INFORMATION