Particularly, in the last 3 years, nine new drugs have received US Food and Drug Administration (FDA) approval for the treatment of AML, including the B-cell lymphoma 2 (BCL-2) inhibitor venetoclax, the isocitrate dehydrogenase (IDH) inhibitors ivosidenib and enasidenib, the FMS-like tyrosine kinase 3 (FLT3) inhibitors midostaurin and gilteritinib, the anti-CD33 monoclonal antibody gemtuzumab ozogamicin (GO), the hedgehog signaling pathway inhibitor glasdegib, a liposomal formulation of a fixed combination of daunorubicin and cytarabine (CPX-351), and the oral HMA CC-486 (refs. 16,20–23). The gene discussed is IDH2; the disease is acute myeloid leukemia.