The major breakthrough involving FLT3-targeted therapy was the phase 3 RATIFY/CALGB 10603 trial of midostaurin, another tyrosine kinase inhibitor (TKI) that targets FLT3, in combination with conventional induction (7+3) chemotherapy for the treatment of newly diagnosed FLT3-mutated AML. The gene discussed is FLT3; the disease is acute myeloid leukemia.