FDA Approves New Option for Acute Myeloid Leukemia

The US Food and Drug administration has recently approved Idhifa (enasidenib) for the treatment of relapsed or refractory acute myeloid leukemia (AML) in adult patients who have IDH2 mutations.

Idhifa is an isocitrate dehydrogenase-2 inhibitor that works by blocking several enzymes associated with promoting cell growth.

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Approval for Idhifa was based on results from a single-arm trial that included 199 patients with relapsed or refractory AML who had IDH2 mutations. After a minimum of 6 months of treatment, 19% of patients experienced complete remission for a median 8.2 months, and 4% experienced complete remission with partial hematologic recovery for a median 9.6 months. Of the 157 patients who required blood or platelet transfusions at baseline, 34% did not require transfusions after receiving Idhifa.

Common adverse effects associated with Idhifa included nausea, vomiting, diarrhea, increased levels of bilirubin, and decreased appetite. Women who are pregnant or breastfeeding are not recommended to receive Idhifa as the drug may harm the developing fetus or newborn baby.

In addition, Idhifa includes a boxed warning for differentiation syndrome. Patients who present with symptoms of differentiation syndrome should be treated with corticosteroids and monitored closely until symptoms abate.

—Melissa Weiss


FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [press release]. FDA. August 1, 2017. Accessed August 1, 2017.