FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Mary L Windle, PharmD


January 11, 2018

In This Article

Idhifa (enasidenib)


Relapsed/refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.


Reversible, selective isocitrate dehydrogenase-2 enzyme (IDH2) inhibitor. Inhibits mutant IDH2 enzyme, which decreases 2-hydroxyglutarate (2-HG) levels and induces myeloid differentiation in vitro.


100 mg PO once daily with or without food until disease progression or unacceptable toxicity.


Approval was based on a phase 1/2 study (AG221-C-001) that enrolled 199 patients with relapsed/refractory AML treated with enasidenib. The median overall survival (OS) with enasidenib was 9.3 months. In those achieving a complete response (CR), the median OS was 19.7 months, and in the non-CR responders, the median OS was 13.8 months. In those without a response, the median OS was 7 months.


Stein EM, et al. Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017 Aug 10;130(6):722-31.


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