The US Food and Drug Administration (FDA) approved enasidenib for adult patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation.1

Enasidenib, the first approved oral isocitrate dehydrogenase-2 inhibitor, also received orphan drug designation and priority review designation from the FDA.

The FDA granted its approval based on evidence from a single-arm phase 2 trial ( Identifier: NCT01915498), which included 199 patients with relapsed or refractory IDH2-positive AML. Patients received oral enasidenib 100mg once daily.2

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After a minimum 6 months of treatment, complete remission (CR) occurred in 19% of patients for a median of 8.2 months, and 4% of patients experienced a complete remission with partial hematologic recovery (CRh) for a median of 9.6 months.

Thirty-four percent of patients who received blood or platelet transfusions prior to enasidenib were able to discontinue transfusion therapy.

Frequently reported adverse effects for enasidenib included nausea, vomiting, diarrhea, decreased appetite, and increased levels of bilirubin.

The FDA also approved the companion diagnostic assay, RealTime IDH2, which is used to identify the IDH2 mutation.

RELATED: Haploidentical Transplantation Effective as Front-line Therapy for Standard-risk ALL

More than 20,000 patients are diagnosed with AML in the US yearly and most patients experience relapse; over 50% of cases result in death.


  1. FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [news release]. Silver Spring, MD: US Food and Drug Administration; Updated August 1, 2017. /PressAnnouncements/ucm569421.htm. Accessed August 1, 2017.
  2. FDA grants approval of IDHIFA, the first oral targeted therapy for adult patients with relapsed/refractory acute myeloid leukemia and an IDH2 mutation [news release]. Summit, NJ and Cambridge, MA: BusinessWire; August 1, 2017. home/20170801006281/en/FDA-Grants-Approval-IDHIFA%C2%AE-Oral-Targeted-Therapy. Accessed August 1, 2017.