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Enasidenib
Source: Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: March 14, 2025 · Captured 2026-09-09.
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Enasidenib is an oral small molecule inhibitor with activity against the altered IDH2 enzyme.
Evidence (enasidenib):
In a phase I/II study, 214 patients with relapsed or refractory AML were treated with enasidenib.[18]
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In a phase I/II study, 214 patients with relapsed or refractory AML were treated with enasidenib.[18]
The overall response rate was 40.3% (95% CI, 33.0%–48.0%), with a median response duration of 5.8 months.
In a phase I/II study, 214 patients with relapsed or refractory AML were treated with enasidenib.[18]
This included 46 patients (26.1%) with a CR or a CR with incomplete hematologic recovery (CRi).
In a phase I/II study, 214 patients with relapsed or refractory AML were treated with enasidenib.[18]
Grade 3 or 4 treatment-related adverse events included indirect hyperbilirubinemia (12%) and differentiation syndrome (7%). As a result, the U.S. Food and Drug Administration (FDA) added a boxed warning to enasidenib, which alerts prescribers that differentiation syndrome can occur.[18][Level of evidence C3]
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Preserved source evidence · Not medical advice