After no substantial innovations in 40 years, the scenario of treatment in acute myeloid leukemia (AML) has recently seen a number of changes, with FDA approval of eight new non-cytostatic compounds.

The new FDA-approved targeted therapies are midostaurin, gilteritinib, glasdegib, ivosidenib, enasidenib, venetoclax, and gemtuzumab ozogamicin.

RATIFY is the first randomized trial to show that the combination of targeted therapy with standard chemotherapy significantly improves survival in AML.

Phase II trials are currently evaluating new drugs targeting EZH2, DOT1L, MLL, BET, and LSD1.