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What is Tibsovo used to treat?

Medically reviewed by Judith Stewart, BPharm. Last updated on June 1, 2022.

Official answer


Tibsovo (ivosidenib) is used to treat acute myeloid leukemia (AML) and cholangiocarcinoma in adult patients who have an isocitrate dehydrogenase-1 (IDH1) genetic mutation as detected by an FDA-approved test.

Mutations in the IDH1 gene can lead to the accumulation of the oncometabolite 2-hydroxyglutarate (2-HG). Increased levels of 2-HG interfere with the processes by which immature blast cells become mature cells.

Tibsovo works by inhibiting the isocitrate dehydrogenase-1 (IDH1) enzyme to decrease abnormal production of 2-HG.

Tibsovo is the first drug approved in the IDH1 inhibitor class.

Acute Myeloid Leukemia (AML)

Acute myeloid leukemia is a type of blood cancer that results in an increased number of abnormal immature white blood cells (myeloblasts) in the bloodstream and bone marrow. People with the IDH1 mutation develop AML because it stops the myeloblasts from becoming healthy white blood cells (granulocytes). Tibsovo is a targeted therapy that specifically targets the cells with the IDH1 mutation and helps the myeloblasts grow into healthy cells. About 6% to 10% of people with AML have an IDH1 mutation.

Tibsovo is indicated for the treatment of adults with IDH1-mutated relapsed or refractory acute myeloid leukemia (AML).

Tibsovo is also indicated with or without azacitidine for adults with newly diagnosed IDH1-mutated AML who are ≥75 years old, or who have comorbidities that preclude the use of intensive induction chemotherapy.


Cholangiocarcinoma is a rare, aggressive cancer of the bile ducts. IDH1 mutations occur in up to 20% of cholangiocarcinoma cases.

Tibsovo is indicated for the treatment of locally advanced or metastatic cholangiocarcinoma in adult patients who have a susceptible IDH1 mutation who have been previously treated.

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