What is Tibsovo used to treat?

Tibsovo (ivosidenib) is used to treat acute myeloid leukemia (AML), cholangiocarcinoma, and myelodysplastic syndromes 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 (when the disease has come back or has not improved after previous treatments).

Tibsovo is also indicated with or without azacitidine for adults with newly diagnosed IDH1-mutated AML who are ≥75 years old, or who have health problems that prevent the use of certain chemotherapy treatments.

Cholangiocarcinoma

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 (bile duct cancer that has spread) in adult patients who have a susceptible IDH1 mutation who have been previously treated.

Relapsed or Refractory Myelodysplastic Syndromes (MDS)

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. Myelodysplastic Syndromes (MDS) are a group of cancers in which immature blood cells in the bone marrow do not mature or become healthy blood cells.

MDS may be characterized by low white or red blood cell counts, low platelet counts, and, in some cases, increased monocytes, a type of white blood cell. Symptoms of this disorder include shortness of breath and feeling tired. Tests that examine the blood and bone marrow can help to diagnose myelodysplastic syndromes.

Tibsovo is indicated for the treatment of adult patients with relapsed or refractory myelodysplastic syndromes (when the disease has come back or has not improved after previous treatments).