Ivosidenib
Ivosidenib is a targeted therapy for acute myeloid leukemia (AML) that specifically inhibits mutated IDH1 enzymes. It is used in adult patients with relapsed or refractory AML whose leukemia cells carry susceptible IDH1 mutations that lead to elevated 2-hydroxyglutarate (2-HG) levels, a metabolite that contributes to malignant cell growth.
Ivosidenib is a targeted therapy for AML patients with specific IDH1 mutations that inhibits the mutant enzyme to reduce harmful 2-HG levels in cancer cells.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- L01XM02
- RxNorm
- 2049873
- Generic
- Ivosidenib
- Class
- atc-l01
- Brands
- Tibsovo
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- tablet
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Treatment of acute myeloid leukemia (AML) in adult patients with relapsed or refractory disease who have susceptible IDH1 mutations. Patient eligibility requires confirmed IDH1 mutation testing prior to treatment initiation.
Ivosidenib is a small molecule inhibitor that selectively targets and inhibits mutant isocitrate dehydrogenase 1 (IDH1) enzymes. By blocking this mutated enzyme, it reduces the overproduction of 2-hydroxyglutarate (2-HG), a metabolite that accumulates in leukemia cells with IDH1 mutations and promotes malignant transformation and differentiation blockade.
Requires confirmed IDH1 mutation status before use. Can cause differentiation syndrome, a potentially serious condition requiring prompt recognition and management. Common side effects may include fatigue, arthralgia, and leukocytosis. Monitor for QT prolongation. May interact with other medications metabolized through similar pathways.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy