Midostaurin, enasidenib, CPX-351, gemtuzumab ozogomycin and venetoclax bring new hope to AML

Andrew H. Wei and Ing S. Tiong


In 2017, four new drugs have so far received FDA marketing approval for the treatment of acute myeloid leukemia (AML), including targeted therapies for mutant FLT3 (midostaurin) and IDH2 (enasidenib), a novel liposomal cytarabine-daunorubicin formulation (CPX-351) for therapy-related AML and AML with myelodysplasia-related changes and finally, resurgence of an antibody-drug conjugate designed to target CD33 (gemtuzumab ozogomycin; GO). Promising results also emerged for the BCL-2 inhibitor venetoclax combined with low-intensity therapy in older patients with AML unfit for intensive chemotherapy. This quintet of new drugs is likely to re-shape the therapeutic landscape of AML.

  • Submitted August 10, 2017.
  • Accepted October 13, 2017.