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Trade namesIdhifa
Other namesEnasidenib mesylate, AG-221
Clinical data
Drug classIsocitrate dehydrogenase 2 inhibitor[1]
Main usesAcute myeloid leukemia (AML)[1]
Side effectsNausea, diarrhea, high bilirubin, lose of appetite[2]
  • AU: D
  • US: N (Not classified yet)
Routes of
By mouth
Typical dose100mg OD[2]
External links
US NLMEnasidenib
License data
Legal status
Chemical and physical data
Molar mass473.383 g·mol−1
3D model (JSmol)
  • CC(C)(CNC1=NC(=NC(=N1)NC2=CC(=NC=C2)C(F)(F)F)C3=NC(=CC=C3)C(F)(F)F)O
  • InChI=InChI=1S/C19H17F6N7O/c1-17(2,33)9-27-15-30-14(11-4-3-5-12(29-11)18(20,21)22)31-16(32-15)28-10-6-7-26-13(8-10)19(23,24)25/h3-8,33H,9H2,1-2H3,(H2,26,27,28,30,31,32)

Enasidenib, sold under the brand name Idhifa, is a medication used to treat acute myeloid leukemia (AML) with mutations of the isocitrate dehydrogenase 2 (IDH2) gene.[1] It is used when other treatments have failed.[2] It is taken by mouth.[2]

Common side effects include nausea, diarrhea, high bilirubin, and lose of appetite.[2] Other side effects may include differentiation syndrome, tumor lysis syndrome, lung problems, infertility, and kidney problems.[1] Use during pregnancy may harm the baby.[2] It works by blocking the action of mutated IDH2.[3]

Enasidenib was approved for medical use in the United States in 2017.[1] It was denied approval in Europe in 2019 due to insufficient evidence of benefit.[3] In the United States a month of treatment costs about 29,500 USD as of 2021.[4]

Medical use

Enasidenib is used to treat relapsed or refractory acute myeloid leukemia in people with specific mutations of the IDH2 gene, determined by an FDA-approved IDH2 companion diagnostic test.[5]


It is taken at a dose of 100 mg per day.[2]

Side effects

The main serious adverse effect of enasidenib is differentiation syndrome.[6]


Isocitrate dehydrogenase is a critical enzyme in the citric acid cycle. Mutated forms of IDH produce high levels of the (R)-enantiomer of 2-hydroxyglutarate (R-2-HG) and can contribute to the growth of tumors. IDH1 catalyzes this reaction in the cytoplasm, while IDH2 catalyzes this reaction in mitochondria. Mutations of IDH2 are more common than IDH1 mutations, 8 to 19% compared to 7 to 14% respectively,[5] in those affected with AML. Enasidenib disrupts this cycle by decreasing total (R)-2-HG levels in the mitochondria.[medical citation needed]


The U.S. Food and Drug Administration (FDA) granted the application for enasidenib fast track designation and orphan drug designation for acute myeloid leukemia in 2014.[6]

Enasidenib was approved by the FDA in August 2017, for relapsed or refractory acute myeloid leukemia (AML) in people with specific mutations of the IDH2 gene, determined by an FDA-approved IDH2 companion diagnostic test.[5][7][8] The FDA considers it to be a first-in-class medication.[9]


  1. 1.0 1.1 1.2 1.3 1.4 "Enasidenib Monograph for Professionals". Drugs.com. Retrieved 15 December 2021.
  2. 2.0 2.1 2.2 2.3 2.4 2.5 2.6 "DailyMed - IDHIFA- enasidenib mesylate tablet, film coated". dailymed.nlm.nih.gov. Retrieved 15 December 2021.
  3. 3.0 3.1 "Idhifa: Withdrawal of the marketing authorisation application". Retrieved 15 December 2021.
  4. "Idhifa Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 15 December 2021.
  5. 5.0 5.1 5.2 Kim ES (October 2017). "Enasidenib: First Global Approval". Drugs. 77 (15): 1705–1711. doi:10.1007/s40265-017-0813-2. PMID 28879540. S2CID 7685848.
  6. 6.0 6.1 Brunton LL, Hilal-Dandan R, Knollmann BC (eds.). Goodman & Gilman's the pharmacological basis of therapeutics (13th ed.). New York. ISBN 9781259584732. OCLC 993810322.
  7. "FDA Approves New Treatment for Leukemia". GEN. August 2, 2017.
  8. "Press release: FDA granted regular approval to enasidenib for the treatment of relapsed or refractory AML". FDA. August 1, 2017.
  9. New Drug Therapy Approvals 2017 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2018. Retrieved 16 September 2020.

External links

External sites: