Based on ALFA-0701 [6] and other clinical trials [7–11], in conjunction with pharmacokinetic/pharmacodynamic modeling to validate the safety and efficacy of the fractionated dosing regimens [13], GO was reapproved by the US FDA in 2017 for the treatment, in combination with standard chemotherapy, of newly-diagnosed CD33 + AML in adults, and as a single agent for relapsed or refractory (R/R) CD33 + AML in adults and pediatric patients ≥ 2 years old [14]. The gene discussed is CD33; the disease is acute myeloid leukemia.