Enasidenib is an isocitrate dehydrogenase-2 inhibitor used to treat relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 mutation. Patients eligible for this treatment are selected by testing the presence of IDH2 mutations in the blood or bone marrow.
- Mechanism
- Curator reviewed · 3 references
Enasidenib is a selective inhibitor of IDH2, a mitochondria-localized enzyme involved in diverse cellular processes, including adaptation to hypoxia, histone demethylation and DNA modification. Wild-type IDH proteins play a cruicial role in the Krebs/citric acid cycle where it catalyzes the oxidative decarboxylation of isocitrate to α-ketoglutarate. In comparison, mutant forms of IDH2 enzyme mediates a neomorphic activity and catalyze reduction of α-KG to the (R) enantiomer of 2-hydroxyglutarate, which is associated with DNA and histone hypermethylation, altered gene expression and blocked cellular differentiation of hematopoietic progenitor cells. Enasidenib primarily targets the mutant IDH2 variants R140Q, R172S, and R172K with higher potency than the wild type enzyme form. Inhibition of the enzyme leads to decreased levels of 2-hydroxyglutarate (2-HG) and promotion of proper differentiation and clonal proliferation of cells of the myeloid lineage.
- Primary indication
- Enasidenib is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.Curator reviewed · 6 structured indications
- Formula / weight
- C19H17F6N7O · 473.383 g/mol (avg)
- First approval
- Canada, 2019 · United States, 2017
- Code names
- AG-221
- Brand names
Resolves to
DYLUUSLLRIQKOE-UHFFFAOYSA-NSMILESCC(C)(O)CNC1=NC(=NC(NC2=CC(=NC=C2)C(F)(F)F)=N1)C1=NC(=CC=C1)C(F)(F)FWhat you can answer from here — as of September 23, 2026
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