ivosidenib at a glance
Medicine class
Mutant IDH1 inhibitor and antineoplastic treatment
Biomarker required
An IDH1 R132 mutation must be confirmed before treatment
Administration
Film-coated tablets swallowed whole with water, without food around administration
Important monitoring
ECGs, blood counts and blood chemistry tests are required before and during treatment
What is ivosidenib used for?
UK-licensed uses are restricted to adults with a confirmed IDH1 R132 mutation.
Newly diagnosed acute myeloid leukaemia, combined with azacitidine, when standard induction chemotherapy is unsuitable.
Previously treated locally advanced or metastatic cholangiocarcinoma, used alone after at least one prior systemic treatment.
How does ivosidenib work?
Mutated IDH1 produces excess 2-hydroxyglutarate, which disrupts normal cell maturation and supports cancer development. Ivosidenib inhibits the mutant enzyme, lowers 2-hydroxyglutarate and helps restore cellular differentiation. Its full anticancer mechanism is not completely understood.
How to take ivosidenib
Follow the specialist cancer team's instructions.
Swallow tablets whole with water.
Do not eat for 2 hours before or 1 hour after taking the tablets.
Avoid grapefruit and grapefruit juice.
Do not swallow the desiccant supplied in the bottle.
If vomiting occurs after administration, do not take replacement tablets.
ivosidenib side effects
Common or expected effects
- AML treatment: vomiting, low neutrophil or platelet counts, raised white-cell count, insomnia, headache, dizziness and musculoskeletal pain.
- Cholangiocarcinoma treatment: fatigue, nausea, abdominal pain, diarrhoea, reduced appetite, abdominal fluid accumulation, vomiting, anaemia, headache, peripheral neuropathy, rash and abnormal liver tests.
Get urgent medical advice
- Differentiation syndrome during AML treatment, including fever, breathlessness, swelling, rapid weight gain, reduced urination or dizziness.
- QT prolongation or serious abnormal heart rhythm, which may cause palpitations, dizziness, light-headedness or fainting.
ivosidenib in pregnancy
Ivosidenib is not recommended during pregnancy because animal studies indicate reproductive toxicity and human data are inadequate. Pregnancy testing is required before treatment where relevant. Patients who could conceive, and males with partners who could conceive, should use effective contraception during treatment and for at least one month afterwards. Hormonal contraception may be less reliable, so a barrier method is also recommended.
ivosidenib while breastfeeding
It is unknown whether ivosidenib enters human milk, and risk to a breastfed infant cannot be excluded. Breastfeeding should stop during treatment and for at least one month afterwards.
ivosidenib interactions
A full interaction review is required before starting ivosidenib and whenever another treatment is added.
Strong CYP3A4 inducers, including rifampicin, carbamazepine, phenytoin, phenobarbital and St John's wort
These may substantially lower ivosidenib exposure and are contraindicated.
Moderate or strong CYP3A4 inhibitors, including clarithromycin, azole antifungals, ritonavir, diltiazem and verapamil
These increase ivosidenib exposure and the risk of QT prolongation, requiring an alternative where possible or specialist management and monitoring.
Medicines that prolong QT
Concurrent use can further increase the risk of dangerous heart-rhythm abnormalities.
Common questions about ivosidenib
Answers are fully visible for fast scanning and source review.
Why is an IDH1 test needed?
Ivosidenib targets mutated IDH1 and is licensed only when an appropriate test confirms an IDH1 R132 mutation.
Is ivosidenib chemotherapy?
It is a targeted anticancer treatment that inhibits mutant IDH1.
Can it be taken with food?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.