FDA Approves First Oral Treatment for Relapsed, Refractory Acute Myeloid Leukemia

Published Online: Tuesday, August 1st, 2017
Officials with the FDA have approved enasidenib (Idhifa, Celgene Corporation) for the treatment relapsed or refractory acute myeloid leukemia (AML) in adult patients who have a specific genetic mutation, isocitrate dehydrogenase-2 (IDH2), according to a FDA press release.

The targeted treatment is approved for the use with a companion diagnostic, the RealTime IDH2 Assay from Abbott Laboratories, which is used to detect specific mutations in the IDH2 gene in patients with AML. Patients who present with IDH2 mutation detections in blood or bone marrow samples may be eligible to be treated with Idhifa.

The approval is based on a single-arm trial of 199 patients with relapsed or refractory AML, who had IDH2 mutations, which demonstrated the drug’s efficacy. Efficacy was measured based on the percentage of patients with no evidence of disease and full recovery of blood counts after treatment (complete remission or CR), as well as patients with no evidence of disease and partial recovery of blood counts after treatment (complete remission with partial hematologic recovery or CRh). Of the patients in the trial who were treated for a minimum of 6 months, 19% experienced CR for an average of 8 months and 4% experienced CRh for an average of 9.6 months.

Of the 157 patients who needed transfusions of blood or platelets at the start of the study, 34% no longer required transfusions after receiving the treatment.

Common adverse effects reported include nausea, vomiting, diarrhea, increased levels of bilirubin, and decreased appetite. Women who are pregnant should not take Idhifa. 


FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm569421.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed August 1, 2017.

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