Extensive research efforts targeting mutant IDH1 culminated in the FDA’s 2022 approval of ivosidenib (synonym: AG-120; brand name: Tibsovo) for a supplemental indication, expanding its use in combination with azacitidine for patients with newly diagnosed IDH1-mutated acute myeloid leukemia (supported by phase III clinical trials such as NCT03173248 and the phase Ib/II trial NCT02677922). Here, IDH1 is linked to acute myeloid leukemia.