The US Food and Drug Administration (FDA) has approved Daiichi Sankyo’s quizartinib (Vanflyta) for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3 internal tandem duplication (ITD)-positive.
It is approved with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy.
The FDA also approved LeukoStrat CDx FLT3 Mutation Assay as a companion diagnostic for Vanflyta.
The efficacy of quizartinib with chemotherapy was evaluated in the QuANTUM-First trial of 539 patients with newly diagnosed FLT3-ITD positive AML. FLT3-ITD status was determined prospectively with a clinical trial assay and verified retrospectively with the companion diagnostic LeukoStrat CDx FLT3 Mutation Assay.
The trial demonstrated a statistically significant improvement in overall survival (OS) for the quizartinib arm. The complete response (CR) rate in the quizartinib arm was 55% with a median duration of 38.6 months and the CR rate in those receiving placebo was 55%, with a median duration of 12.4 months.
“The approval of Vanflyta 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 Erba, Professor of Medicine, Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke Cancer Institute. “In the QuANTUM-First trial, Vanflyta added to standard chemotherapy and continued as maintenance resulted in longer remission and prolonged overall survival and it will be a much-needed new treatment option that has potential to change the way FLT3-ITD positive AML is treated.”
Quizartinib is available only through a restricted programme under a Risk Evaluation and Mitigation Strategy (REMS), called the Vanflyta REMS.
This application was granted priority review, fast track designation and orphan drug designation.