Tibsovo: Uses, Dosage & Side Effects
A targeted IDH1 inhibitor for IDH1-mutated acute myeloid leukemia (AML) and locally advanced or metastatic cholangiocarcinoma (bile duct cancer)
Tibsovo (ivosidenib) is a targeted oral anticancer medication that works by inhibiting the mutated isocitrate dehydrogenase 1 (IDH1) enzyme. It is approved for the treatment of acute myeloid leukemia (AML) with an IDH1 R132 mutation in combination with azacitidine, and for locally advanced or metastatic cholangiocarcinoma (bile duct cancer) with an IDH1 R132 mutation in patients who have received at least one prior line of systemic therapy. An IDH1 mutation test must be performed before starting treatment. Tibsovo is taken as an oral tablet, 500 mg once daily on an empty stomach. Important safety considerations include the risk of differentiation syndrome in AML patients and QTc prolongation requiring regular ECG monitoring.
Quick Facts: Tibsovo
Key Takeaways
- Tibsovo (ivosidenib) is a targeted therapy that selectively inhibits the mutated IDH1 enzyme, reducing abnormal levels of the oncometabolite 2-hydroxyglutarate (2-HG) and restoring normal cell differentiation in IDH1-mutated cancers.
- It is approved for two distinct indications: IDH1-mutated AML (in combination with azacitidine) for patients not eligible for intensive chemotherapy, and IDH1-mutated locally advanced or metastatic cholangiocarcinoma after at least one prior systemic therapy.
- An IDH1 mutation test using a validated assay is mandatory before initiating treatment; Tibsovo should only be prescribed for patients confirmed to have an IDH1 R132 mutation.
- Serious risks include differentiation syndrome (in AML patients, occurring up to 46 days after treatment start) and QTc prolongation, which requires ECG monitoring before treatment, weekly for the first 3 weeks, and then monthly.
- Tibsovo must not be used with dabigatran, St John’s wort, rifampicin, carbamazepine, phenobarbital, or phenytoin, and grapefruit or grapefruit juice must be avoided during treatment; effective non-hormonal contraception is required as the drug may reduce the effectiveness of hormonal contraceptives.
What Is Tibsovo and What Is It Used For?
Tibsovo contains the active substance ivosidenib, a first-in-class small molecule inhibitor that specifically targets the mutated form of the isocitrate dehydrogenase 1 (IDH1) enzyme. IDH1 is a metabolic enzyme that, in its normal form, plays a key role in cellular energy metabolism by converting isocitrate to alpha-ketoglutarate. However, when the IDH1 gene acquires certain mutations (specifically at the R132 position), the enzyme gains an abnormal function: it produces excessive amounts of a metabolite called 2-hydroxyglutarate (2-HG).
Elevated 2-HG levels have profound effects on cell biology. This oncometabolite interferes with enzymes that regulate gene expression through epigenetic modifications, particularly those involved in DNA and histone demethylation. The result is a block in normal cellular differentiation – cells that should mature into functional blood cells (in the case of AML) or normal bile duct cells (in the case of cholangiocarcinoma) instead remain in an immature, rapidly dividing state, driving tumor growth. By selectively inhibiting the mutant IDH1 enzyme, ivosidenib reduces 2-HG levels back to near-normal, which removes the differentiation block and allows malignant cells to resume normal maturation and eventually undergo programmed cell death.
IDH1 R132 mutations are found in approximately 6–10% of patients with acute myeloid leukemia and in about 13–20% of patients with intrahepatic cholangiocarcinoma (the most common form of bile duct cancer). Identification of this mutation through a validated diagnostic test is a prerequisite for treatment with Tibsovo. The test is typically performed on a bone marrow aspirate or peripheral blood sample for AML, or on a tumor tissue biopsy for cholangiocarcinoma.
Tibsovo is approved by regulatory authorities including the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) for the following indications:
- Acute myeloid leukemia (AML) with IDH1 R132 mutation: Tibsovo is used in combination with azacitidine for the treatment of adult patients with newly diagnosed AML who carry an IDH1 R132 mutation and who are not eligible for standard intensive induction chemotherapy. The pivotal AGILE trial demonstrated significantly improved event-free survival, overall survival, and complete remission rates with the combination of ivosidenib plus azacitidine compared with placebo plus azacitidine.
- Cholangiocarcinoma with IDH1 R132 mutation: Tibsovo is used as monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma (bile duct cancer) harboring an IDH1 R132 mutation who have received at least one prior line of systemic therapy. The ClarIDHy trial demonstrated improved progression-free survival with ivosidenib compared with placebo, with a clinically meaningful benefit also observed in overall survival after adjusting for crossover from the placebo arm.
AML is an aggressive cancer of the blood and bone marrow characterized by the rapid proliferation of abnormal white blood cells (blasts) that interfere with the production of normal blood cells. Cholangiocarcinoma is a relatively rare but often aggressive cancer arising from the epithelial cells lining the bile ducts. Both cancers have historically had limited treatment options, and the identification of targetable IDH1 mutations has opened a new avenue of precision medicine for these patients.
Tibsovo represents a precision medicine approach to cancer treatment. It is only effective in patients whose tumors carry a specific IDH1 R132 mutation. Before starting treatment, your doctor will order a validated mutation test to confirm that this mutation is present. If the mutation is not detected, Tibsovo will not be prescribed, as it is unlikely to provide benefit in IDH1 wild-type (non-mutated) tumors.
What Should You Know Before Taking Tibsovo?
Contraindications
There are specific situations in which Tibsovo must not be used. Understanding these absolute contraindications is essential before starting treatment.
- Hypersensitivity: Do not take Tibsovo if you are allergic to ivosidenib or any of the other ingredients in the tablet (including lactose).
- Concurrent use with dabigatran: Tibsovo must not be used together with dabigatran (a blood-thinning medicine), as ivosidenib can significantly alter dabigatran levels in the body, leading to an increased risk of bleeding or reduced effectiveness.
- Concurrent use with strong CYP3A4 inducers: St John’s wort (a herbal remedy), rifampicin, carbamazepine, phenobarbital, and phenytoin must not be taken with Tibsovo, as they can drastically reduce ivosidenib levels and make the treatment ineffective.
- Congenital long QT syndrome: Patients with this inherited heart rhythm condition must not take Tibsovo.
- Family history of sudden cardiac death or significant arrhythmias: If you have a family history of sudden cardiac death, unexplained syncope (fainting), or clinically significant cardiac arrhythmias, Tibsovo is contraindicated.
- Severe QTc prolongation: If you already have severe prolongation of the QTc interval on your ECG before starting treatment, Tibsovo must not be used.
Warnings and Precautions
Differentiation syndrome is a potentially life-threatening complication that can occur in AML patients treated with Tibsovo, typically within the first 46 days of treatment but sometimes later. It occurs when leukemia cells rapidly mature (differentiate), releasing inflammatory substances into the bloodstream. Symptoms include unexplained fever, difficulty breathing, rapid weight gain, swelling (edema), low blood pressure, acute kidney injury, and fluid buildup around the lungs or heart. If you experience any of these symptoms, contact your doctor immediately. Treatment with corticosteroids must be started promptly, and Tibsovo may need to be temporarily interrupted.
Tibsovo can cause prolongation of the QTc interval, a change in the heart’s electrical activity measured on an ECG. Severe QTc prolongation can lead to dangerous heart rhythm disturbances (ventricular tachycardia, torsades de pointes) and, in extreme cases, sudden cardiac death. Tibsovo must not be started if the baseline QTc interval is already severely prolonged. If QTc exceeds 500 milliseconds during treatment, Tibsovo must be temporarily stopped and can only be restarted at a reduced dose once the QTc returns to acceptable levels.
Before and during treatment with Tibsovo, discuss the following with your doctor:
- Electrolyte imbalances: Low levels of potassium, magnesium, or calcium can increase the risk of QTc prolongation. Your doctor will check and correct any electrolyte abnormalities before and during treatment.
- Other QT-prolonging medications: Inform your doctor about all other medicines you are taking, as many drugs can also prolong the QTc interval, and the effects may be additive.
- Leukocytosis (high white blood cell count): In AML patients, Tibsovo can cause a temporary increase in white blood cells (leukocytosis), which is sometimes part of differentiation syndrome. Your doctor will monitor blood counts regularly.
- Guillain-Barré syndrome: Very rarely, Tibsovo treatment has been associated with Guillain-Barré syndrome, a serious condition affecting the peripheral nerves that causes muscle weakness and sometimes paralysis. Seek medical attention immediately if you develop progressive weakness in the legs, arms, or face, or difficulty breathing.
Regular Monitoring
While taking Tibsovo, your doctor will schedule regular monitoring appointments that include:
- ECG monitoring: Before starting treatment, then weekly during the first 3 weeks, and monthly thereafter. Additional ECGs may be performed if you start or stop other medications that may affect the QTc interval, or if you develop relevant symptoms.
- Blood tests: Regular complete blood counts (especially important in AML), electrolytes (potassium, magnesium, calcium), and liver function tests.
- Clinical assessments: Monitoring for signs and symptoms of differentiation syndrome (AML patients), particularly during the first 46 days of treatment.
Pregnancy and Breastfeeding
Tibsovo is not recommended during pregnancy. Based on its mechanism of action and findings from animal studies, ivosidenib may cause harm to the developing fetus. Women of childbearing potential must have a pregnancy test before starting treatment and use effective contraception during treatment and for at least 1 month after the last dose.
An important consideration is that ivosidenib may reduce the effectiveness of hormonal contraceptives (such as the pill, patches, or implants) by inducing enzymes that break them down. Therefore, women using hormonal contraception must also use a barrier method (such as condoms) or switch to a non-hormonal method of contraception. Discuss the most appropriate contraceptive options with your doctor.
Men taking Tibsovo should also use effective contraception during treatment and for at least 1 month after the last dose. It is not known whether ivosidenib or its metabolites are excreted in human breast milk. Because of the potential for serious adverse effects in a breastfed infant, breastfeeding is not recommended during treatment and for at least 1 month after the last dose.
Driving and Operating Machinery
Tibsovo may have a minor effect on the ability to drive and use machines. Dizziness and fatigue have been reported as side effects. If you experience these symptoms, do not drive or operate machinery until they have resolved. Exercise caution and assess your fitness to perform these activities based on how you feel during treatment.
Important Information About Ingredients
Tibsovo tablets contain lactose. If you have been told by your doctor that you have an intolerance to some sugars, contact your doctor before taking this medicine.
Each tablet also contains less than 1 mmol (23 mg) of sodium, meaning it is essentially sodium-free. This is relevant for patients on a controlled sodium diet.
How Does Tibsovo Interact with Other Drugs?
Ivosidenib is primarily metabolized by the CYP3A4 enzyme and is also a substrate of the P-glycoprotein (P-gp) transporter. Additionally, ivosidenib itself acts as a moderate inducer of CYP3A4 and an inhibitor of certain drug transporters. This complex pharmacological profile means it can both be affected by other drugs and affect the levels of co-administered medications. It is essential to inform your doctor about all medicines, herbal products, and supplements you are taking.
Contraindicated Combinations
| Interacting Drug | Effect | Clinical Significance |
|---|---|---|
| Dabigatran | Ivosidenib inhibits the P-gp transporter, significantly increasing dabigatran exposure and bleeding risk | Absolute contraindication – never combine |
| St John’s wort (Hypericum perforatum) | Strong CYP3A4 inducer that drastically reduces ivosidenib levels | Absolute contraindication – renders treatment ineffective |
| Rifampicin | Strong CYP3A4 inducer that drastically reduces ivosidenib levels | Absolute contraindication – renders treatment ineffective |
| Carbamazepine | Strong CYP3A4 inducer that drastically reduces ivosidenib levels | Absolute contraindication – renders treatment ineffective |
| Phenobarbital | Strong CYP3A4 inducer that drastically reduces ivosidenib levels | Absolute contraindication – renders treatment ineffective |
| Phenytoin | Strong CYP3A4 inducer that drastically reduces ivosidenib levels | Absolute contraindication – renders treatment ineffective |
Major Interactions
| Interacting Drug | Effect | Clinical Significance |
|---|---|---|
| Strong CYP3A4 inhibitors (e.g., itraconazole, ketoconazole, posaconazole, voriconazole, clarithromycin) | Increased ivosidenib plasma levels, potentially increasing the risk of QTc prolongation and other toxicities | Avoid if possible; if unavoidable, reduce Tibsovo dose to 250 mg daily and monitor ECG closely |
| Hormonal contraceptives (combined pill, patch, implant) | Ivosidenib induces CYP3A4, which may reduce hormonal contraceptive levels and effectiveness | Use additional barrier method or switch to non-hormonal contraception |
| QTc-prolonging medications (e.g., ondansetron, fluoroquinolones, antiarrhythmics) | Additive QTc prolongation, increased risk of cardiac arrhythmias | Use with extreme caution; more frequent ECG monitoring required |
| Sensitive CYP3A4 substrates (e.g., midazolam, simvastatin, amlodipine, ciclosporin) | Ivosidenib moderately induces CYP3A4, reducing levels of these drugs | May require dose adjustment of the co-administered drug; monitor effectiveness |
Other Interactions
| Interacting Drug | Effect | Clinical Significance |
|---|---|---|
| Moderate CYP3A4 inhibitors (e.g., fluconazole, erythromycin, diltiazem) | Modest increase in ivosidenib levels | Use with caution; monitor for increased side effects and ECG changes |
| Moderate CYP3A4 inducers (e.g., efavirenz, bosentan, modafinil) | Modest decrease in ivosidenib levels | Use with caution; efficacy may be reduced |
| P-gp substrates (e.g., digoxin, fexofenadine) | Ivosidenib inhibits P-gp, potentially increasing levels of these drugs | Monitor for increased side effects of the co-administered drug |
| BCRP substrates (e.g., rosuvastatin, methotrexate) | Ivosidenib inhibits BCRP transporter, potentially increasing levels of these drugs | Monitor for toxicity; dose reduction of co-administered drug may be needed |
Grapefruit and grapefruit juice: Grapefruit is a moderate CYP3A4 inhibitor and must be avoided during treatment with Tibsovo. Consuming grapefruit or grapefruit juice can increase ivosidenib blood levels, raising the risk of side effects including QTc prolongation. Seville (bitter) oranges and starfruit should also be avoided for the same reason.
What Is the Correct Dosage of Tibsovo?
Tibsovo is an oral medication taken at home. The tablets should be swallowed whole with water at approximately the same time each day. Adherence to the fasting requirement is important, as food increases the absorption of ivosidenib and may alter its safety profile. Your doctor will confirm the diagnosis with an IDH1 mutation test before prescribing treatment.
Dosing for Acute Myeloid Leukemia (AML)
Tibsovo + Azacitidine for IDH1-Mutated AML
Dose: 500 mg (2 tablets) orally once daily
Timing: On an empty stomach – no food for at least 2 hours before and 1 hour after
Combination: Given together with azacitidine (75 mg/m² subcutaneously or intravenously on Days 1–7 of each 28-day cycle, or in an alternative schedule as determined by your doctor)
Duration: Treatment continues until disease progression, unacceptable toxicity, or until your doctor determines that it should be stopped. In the AML indication, a minimum treatment duration of 6 months is generally recommended to assess response, as some patients achieve remission only after several cycles.
Dosing for Cholangiocarcinoma
Tibsovo Monotherapy for IDH1-Mutated Cholangiocarcinoma
Dose: 500 mg (2 tablets) orally once daily
Timing: On an empty stomach – no food for at least 2 hours before and 1 hour after
Duration: Treatment continues until disease progression or unacceptable toxicity
Dose Reductions
Your doctor may reduce the dose to 250 mg (1 tablet) once daily in the following situations:
- QTc prolongation: If the QTc interval exceeds 500 milliseconds on your ECG, Tibsovo will be temporarily stopped and restarted at 250 mg once the QTc returns to acceptable levels.
- Strong CYP3A4 inhibitors: If you need to take a strong CYP3A4 inhibitor (such as certain antifungal medications) concurrently, the Tibsovo dose should be reduced to 250 mg daily.
- Other toxicities: For certain side effects that do not resolve with supportive care, your doctor may reduce the dose or temporarily interrupt treatment.
Do not take more or less than the prescribed dose without consulting your doctor. If you have difficulty swallowing tablets, discuss alternatives with your healthcare team. Do not crush, chew, or break the tablets.
Missed Dose
If you miss a dose of Tibsovo, or if you vomit after taking a dose, do not take an extra dose. Simply take your next dose at the usual scheduled time. Do not take a double dose to make up for a missed one. If you miss several doses or are unsure about what to do, contact your doctor or pharmacist for advice.
Overdose
If you accidentally take more Tibsovo than prescribed, contact your doctor or go to the nearest hospital emergency department immediately. There is no specific antidote for ivosidenib overdose. Treatment would be supportive, with particular attention to monitoring the QTc interval on ECG, as the risk of QTc prolongation may be increased at higher doses. Bring the medicine package with you so that the medical team knows what you have taken.
What Are the Side Effects of Tibsovo?
Like all medicines, Tibsovo can cause side effects, although not everyone will experience them. The type and frequency of side effects differ between the two approved indications (AML and cholangiocarcinoma). Your medical team will monitor you regularly and help manage any side effects that occur.
Side Effects in AML Patients (Tibsovo + Azacitidine)
Very Common
May affect more than 1 in 10 people
- Vomiting
- Neutropenia (decreased white blood cells / neutrophils)
- Thrombocytopenia (decreased platelets)
- Leukocytosis (increased white blood cells)
- Insomnia (difficulty sleeping)
- Pain in extremities (arms or legs)
- Joint pain (arthralgia)
- Headache
- Dizziness
- Back pain
Common
May affect up to 1 in 10 people
- Mouth and throat pain (oropharyngeal pain)
- Peripheral neuropathy (numbness, tingling in hands/feet)
- Leukopenia (decreased total white blood cells)
- Differentiation syndrome
- QTc prolongation on ECG
Side Effects in Cholangiocarcinoma Patients
Very Common
May affect more than 1 in 10 people
- Fatigue (tiredness)
- Nausea
- Abdominal pain
- Diarrhea
- Decreased appetite
- Ascites (fluid buildup in the abdomen)
- Vomiting
- Anemia (low red blood cells)
- Headache
- Elevated AST (liver enzyme)
- Peripheral neuropathy (numbness, tingling)
- Rash
- Elevated bilirubin (jaundice marker)
Common
May affect up to 1 in 10 people
- Decreased white blood cell count
- Decreased platelet count
- Elevated ALT (liver enzyme)
- Falls
- Hyperbilirubinemia (high bilirubin in blood)
- Cholestatic jaundice (bile flow obstruction causing yellowing)
- QTc prolongation on ECG
Differentiation syndrome is a serious and potentially fatal complication specific to the AML indication. It can occur at any time during the first 46 days of treatment, but sometimes later. Symptoms include unexplained fever, difficulty breathing, low blood pressure, rapid weight gain, swelling, pulmonary infiltrates on imaging, and signs of fluid retention including pleural or pericardial effusions. If you experience any of these symptoms, seek immediate medical attention. Early recognition and treatment with corticosteroids is critical for a good outcome.
QTc prolongation is a cardiac safety concern that applies to both AML and cholangiocarcinoma patients taking Tibsovo. This effect on the heart’s electrical activity can potentially lead to serious arrhythmias. Report any symptoms such as palpitations, dizziness, lightheadedness, fainting, or seizures to your doctor immediately. Regular ECG monitoring is essential throughout treatment.
If you experience any side effects, including those not listed here, tell your doctor, pharmacist, or nurse. You can also report suspected side effects to your national pharmacovigilance authority (e.g., the EMA in Europe, the FDA MedWatch program in the United States, or the MHRA Yellow Card Scheme in the United Kingdom) to help monitor the ongoing safety profile of Tibsovo.
How Should You Store Tibsovo?
Proper storage of Tibsovo ensures the tablets remain effective and safe throughout the treatment period.
- Temperature: No special temperature storage requirements. Store at room temperature.
- Moisture protection: Keep the bottle tightly closed at all times to protect the tablets from moisture. The bottle contains a desiccant (drying agent) – do not remove it or swallow it.
- Light: No special light protection requirements.
- Expiry: Do not use after the expiry date stated on the bottle label and carton after EXP. The expiry date refers to the last day of that month.
- Children: Keep out of the sight and reach of children.
Do not dispose of unused tablets via household waste or wastewater. Ask your pharmacist about how to properly dispose of medicines you no longer need. This will help protect the environment.
What Does Tibsovo Contain?
Active Substance
The active substance is ivosidenib. Each film-coated tablet contains 250 mg of ivosidenib. The standard daily dose of 500 mg is achieved by taking 2 tablets.
Inactive Ingredients (Excipients)
The tablet core contains:
- Microcrystalline cellulose
- Croscarmellose sodium
- Hypromellose acetate succinate
- Colloidal anhydrous silica
- Magnesium stearate
- Sodium laurilsulfate
- Lactose monohydrate
The film coating contains:
- Hypromellose
- Titanium dioxide (E171)
- Lactose monohydrate
- Macrogol
- Triacetin
- Indigo carmine aluminium lake (E132)
Appearance and Packaging
Tibsovo tablets are blue, oval, film-coated tablets with “IVO” debossed on one side and “250” on the other side. Each high-density polyethylene (HDPE) bottle contains 60 tablets and a desiccant canister. The bottle is sealed with a child-resistant closure.
Manufacturer
Tibsovo is marketed by Les Laboratoires Servier (Suresnes, France). It was originally developed by Agios Pharmaceuticals (now part of Servier). The marketing authorization holder for the European Union is Les Laboratoires Servier.
Frequently Asked Questions About Tibsovo
Tibsovo (ivosidenib) is used for two main indications: (1) treatment of acute myeloid leukemia (AML) with an IDH1 R132 mutation, in combination with azacitidine, in adult patients not eligible for standard intensive chemotherapy; and (2) treatment of locally advanced or metastatic cholangiocarcinoma (bile duct cancer) with an IDH1 R132 mutation in adults who have received at least one prior line of systemic therapy. A validated IDH1 mutation test is required before starting treatment.
Tibsovo is taken as 2 tablets (500 mg total) by mouth once daily. It must be taken on an empty stomach – no food should be consumed for at least 2 hours before and 1 hour after taking the tablets. Swallow the tablets whole with water; do not crush, chew, or break them. Take them at approximately the same time each day. Grapefruit and grapefruit juice must be avoided during treatment.
Differentiation syndrome is a potentially serious complication that can occur in AML patients (not cholangiocarcinoma patients) treated with Tibsovo, usually within the first 46 days of treatment. It happens when leukemia cells rapidly mature, releasing inflammatory substances. Symptoms include unexplained fever, difficulty breathing, fluid retention, rapid weight gain, low blood pressure, and organ dysfunction. While it can be serious, early recognition and treatment with corticosteroids leads to good outcomes in most cases. Your medical team will monitor you closely during the early weeks of treatment.
Tibsovo can cause prolongation of the QTc interval on the ECG, which is a measure of the heart’s electrical recovery time. If the QTc interval becomes too long, it can lead to potentially dangerous heart rhythm disturbances. Regular ECG monitoring – before treatment, weekly during the first 3 weeks, and then monthly – allows your doctor to detect any significant QTc changes early and take appropriate action, such as temporarily stopping the medication or adjusting the dose.
Tibsovo has several important drug interactions. It must never be taken with dabigatran, St John’s wort, rifampicin, carbamazepine, phenobarbital, or phenytoin. Many other medications may also interact with Tibsovo. It is essential to tell your doctor about all medicines, herbal products, and supplements you take, including over-the-counter products. Your doctor will review your medication list and make any necessary changes before starting Tibsovo. Also avoid grapefruit and grapefruit juice during treatment.
No, Tibsovo is not recommended during pregnancy or breastfeeding. Based on its mechanism of action, it may harm the developing fetus. Women of childbearing potential must use effective contraception during treatment and for at least 1 month after the last dose. Importantly, Tibsovo may reduce the effectiveness of hormonal contraceptives (the pill, patches, implants), so an additional barrier method or a switch to non-hormonal contraception is necessary. Men should also use effective contraception. Breastfeeding is not recommended during treatment and for 1 month after the last dose.
References
- European Medicines Agency (EMA). Tibsovo (ivosidenib) – Summary of Product Characteristics. Last updated 2025. Available from: EMA EPAR.
- U.S. Food and Drug Administration (FDA). Tibsovo (ivosidenib) Prescribing Information. Revised 2024. Available from: FDA Drug Label.
- Montesinos P, Recher C, Vives S, et al. Ivosidenib and Azacitidine in IDH1-Mutated Acute Myeloid Leukemia (AGILE). N Engl J Med. 2022;386(16):1519–1531. doi:10.1056/NEJMoa2117344.
- Abou-Alfa GK, Macarulla T, Javle MM, et al. Ivosidenib in IDH1-Mutant, Chemotherapy-Refractory Cholangiocarcinoma (ClarIDHy): A Multicentre, Randomised, Double-Blind, Placebo-Controlled, Phase 3 Study. Lancet Oncol. 2020;21(6):796–807. doi:10.1016/S1470-2045(20)30157-1.
- Zhu AX, Macarulla T, Javle MM, et al. Final Overall Survival Efficacy Results of Ivosidenib for Patients with Advanced Cholangiocarcinoma with IDH1 Mutation: The Phase 3 Randomized Clinical ClarIDHy Trial. JAMA Oncol. 2021;7(11):1669–1677. doi:10.1001/jamaoncol.2021.3836.
- National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology: Acute Myeloid Leukemia. Version 3.2025.
- ESMO Clinical Practice Guidelines. Biliary Tract Cancer: ESMO Clinical Practice Guideline for Diagnosis, Treatment and Follow-Up. Ann Oncol. 2024.
- World Health Organization (WHO). WHO Model List of Essential Medicines – 24th List. Geneva: WHO; 2025.
- DiNardo CD, Stein EM, de Botton S, et al. Durable Remissions with Ivosidenib in IDH1-Mutated Relapsed or Refractory AML. N Engl J Med. 2018;378(25):2386–2398. doi:10.1056/NEJMoa1716984.
- Lowery MA, Burris HA, Janku F, et al. Safety and Activity of Ivosidenib in Patients with IDH1-Mutant Advanced Cholangiocarcinoma: A Phase 1 Study. Lancet Gastroenterol Hepatol. 2019;4(9):711–720. doi:10.1016/S2468-1253(19)30189-X.
Editorial Team
This article was written and reviewed by the iMedic Medical Editorial Team, comprising licensed specialist physicians with expertise in oncology, hematology, and clinical pharmacology.
Medical Content
iMedic Oncology Editorial Team – specialist physicians in hematology/oncology with clinical experience in AML and biliary tract cancer treatment
Medical Review
iMedic Medical Review Board – independent panel verifying accuracy against EMA SmPC, FDA label, NCCN, and ESMO guidelines
Pharmacology Review
iMedic Clinical Pharmacology Team – specialists in drug interactions, pharmacokinetics, and medication safety
Accessibility & SEO
iMedic Digital Health Team – ensuring WCAG 2.2 AAA compliance and optimal search visibility
All content follows the GRADE evidence framework and is reviewed according to international medical guidelines. iMedic receives no commercial funding from pharmaceutical companies.