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IDH1 Inhibitors: Uses, Common Brands, and Safety Info

IDH1 inhibitors are a class of drugs used to treat certain types of cancer, including acute myeloid leukemia (AML) and cholangiocarcinoma. Common brands include Ivosidenib and Enasidenib. These medications target and inhibit the IDH1 enzyme, reducing cancer cell growth. Side effects may include nausea, vomiting, and muscle pain. Close monitoring by healthcare professionals is important.

IDH1 Inhibitors

IDH1 (Isocitrate Dehydrogenase 1) inhibitors are a class of drugs used in the treatment of certain types of cancers. These medications work by targeting and inhibiting the activity of the IDH1 enzyme, which plays a crucial role in cellular metabolism and the development of cancer.


IDH1 inhibitors are primarily used in the treatment of acute myeloid leukemia (AML) and cholangiocarcinoma, which is a form of bile duct cancer. These drugs are particularly beneficial for patients with specific genetic mutations involving the IDH1 enzyme.

In patients with AML, IDH1 mutations are associated with a poorer prognosis, and IDH1 inhibitors, such as ivosidenib and enasidenib, have shown promising results in improving patient outcomes. These medications can effectively target and inhibit the mutated IDH1 enzyme, leading to a decrease in the production of oncometabolite 2-hydroxyglutarate (2-HG) and a subsequent reduction in cancer cell growth.

Common Brands

Some common IDH1 inhibitors include:

  • Ivosidenib (Tibsovo)

  • Enasidenib (Idhifa)


As with any medication, IDH1 inhibitors may cause certain side effects. Common side effects include nausea, vomiting, diarrhea, fatigue, decreased appetite, and muscle pain. In some cases, these drugs may also be associated with more serious adverse events such as differentiation syndrome, an IDH1 inhibitor-specific side effect characterized by fever, respiratory symptoms, and fluid retention.

It is important for patients taking IDH1 inhibitors to communicate with their healthcare providers about any side effects experienced, as supportive treatments and dose adjustments may be necessary. Additionally, IDH1 inhibitors may interact with other medications, so it's crucial to inform the healthcare team about all prescribed and over-the-counter drugs, as well as any herbal or dietary supplements.

Overall, IDH1 inhibitors have shown promising results in the treatment of specific types of cancers, particularly AML and cholangiocarcinoma. However, individual responses to these medications may vary, and close monitoring by healthcare professionals is essential to ensure optimal safety and efficacy.

List of IDH1 Inhibitors