The U.S. Food and Drug Administration (FDA) approved olutasidenib for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible IDH1 mutation in adult patients. The mutation must be detected by the Abbott RealTime IDH1 Assay, an FDA-approved test.
Approval was based on the results of an open-label, single-arm, multicenter trial, dubbed Study 2102-HEM-101. Of 147 patients, the rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 35% with a median duration of 25.9 months. Of the 35% who achieved CR + CRh, 92% achieved CR with a median duration of response of 28.1 months.