Olutasidenib for Adults with Relapsed or Refractory Acute Myeloid Leukaemia with Isocitrate Dehydrogenase 1 Mutation

Management of patients with relapsed or refractory (R/R) acute myeloid leukaemia (AML) is challenging, as their prognosis remains poor with conventional therapies, underlining the need for the development of new drugs. Isocitrate dehydrogenase (IDH) inhibitors are targeted therapies that have been developed over several years for the treatment of AML. This review will focus on olutasidenib, a selective, oral, small-molecule inhibitor of the mutant IDH1 enzyme, approved by the US Food and Drug Administration (FDA) on 01 December 2022 for adult patients with R/R AML with IDH1 mutation detected by an FDA-approved test. We will review the mechanisms of IDH1 mutations in AML, inhibition of mutations by olutasidenib and the clinical efficacy and safety of olutasidenib in R/R AML.