The U.S. Food and Drug Administration (FDA) approved quizartinib for the treatment of newly diagnosed FLT3-ITD-positive acute myeloid leukemia (AML) in adult patients. The indication approves quizartinib in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, as well as maintenance monotherapy after consolidation chemotherapy.
Quizartinib is an orally administered, type-II FLT3 inhibitor that works by selectively targeting FLT3-ITD mutations. The FDA granted priority review of the treatment and fast track designation for the indication.
Approval was based on the results of a randomized, double-blind, placebo-controlled trial dubbed QuANTUM-First. Results found a statistically significant improvement in overall survival (OS) among patients who received quizartinib compared to those who received placebo.
“The approval of [quizartinib] represents a significant advancement for the treatment of patients with newly diagnosed FLT3-ITD-positive AML, which is one of the most aggressive and difficult-to-treat subtypes,” said Harry P. Erba, MD, PhD, professor of medicine in the Division of Hematologic Malignancies and Cellular Therapy at Duke Cancer Institute. “In the QuANTUM-First trial, [quizartinib] added to standard chemotherapy and continued as maintenance resulted in longer remission and prolonged [OS], and it will be a much-needed new treatment option that has potential to change the way FLT3-ITD-positive AML is treated.”
Currently, quizartinib is also approved in Japan for adult patients with FLT3-ITD-positive AML, in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy. It is also approved as maintenance monotherapy for adult patients with newly diagnosed FLT3-ITD-positive AML and as a monotherapy for relapsed/refractory AML that is FLT3-ITD positive.