An oral, small molecule inhibitor of mutant isocitrate dehydrogenase 2 (IDH2) enzymes.

IDH2 mutations occur in approximately 15% of patients with acute myelogenous leukemia.

The mutant proteins catalyze production of“ metabolite 2+hydroxyglutarate, resulting in downstream epigenetic effects related to the growth and proliferation of AML, including hypermethylation and blocks differentiation of hematopoietic cells.

It inhibits mutant IDH2 enzymes and results in decreased 2-hydroxyglutarate levels and differentiation of leukemic cells.

Trade name Idhifa.

In patients with relapsed or refractory IDH2 mutated AML undergo durable responses including complete remission in about 20% of patients.

May be associated with rapid escalation of blood counts and may be associated with fever, shortness of breath, and low oxygen levels.

Side effects include differentiation syndrome, nausea, vomiting, diarrhea, impaired appetite and infections.

Responses to AML can take many weeks, and the drug needs to be continued for at least 8 to 12 weeks to allow a response to develop.

100 mg/D orally.

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