A randomized phase III trial (RATIFY study, NCT00651261) showed that the addition of midostaurin to cytarabine chemotherapy significantly improved overall survival (OS) for FLT3-mutated AML patients.104 Based on the beneficial results of the RATIFY study, midostaurin was approved by the US FDA for combination therapy with standard chemotherapy in 2017 (Table 1). This evidence concerns the gene FLT3 and acute myeloid leukemia.