FDA approves quizartinib for newly diagnosed acute myeloid leukemia
Regulatory approval: On July 20, 2023, the FDA approved quizartinib (Vanflyta) in combination with standard cytarabine/anthracycline induction and cytarabine consolidation, and as post-consolidation maintenance monotherapy, for adults with newly diagnosed FLT3-ITD–positive AML as detected by an FDA-approved test. The companion diagnostic LeukoStrat CDx FLT3 Mutation Assay was also approved. Approval was supported by QuANTUM-First, which showed an overall survival benefit versus placebo (HR 0.78; 95% CI 0.62–0.98).
FDA approval: quizartinib for newly diagnosed FLT3-ITD–positive AML (July 20, 2023)
On July 20, 2023, the US FDA granted regulatory approval to quizartinib (Vanflyta; Daiichi Sankyo, Inc.) for adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 internal tandem duplication (ITD)-positive, as detected by an FDA-approved test. The approved regimen is quizartinib with standard cytarabine and anthracycline induction and cytarabine consolidation, followed by maintenance monotherapy after consolidation chemotherapy.
The FDA also approved the LeukoStrat CDx FLT3 Mutation Assay as a companion diagnostic for Vanflyta.
Supporting evidence: Efficacy was evaluated in QuANTUM-First (NCT02668653), a randomized, double-blind, placebo-controlled trial (N=539) in newly diagnosed FLT3-ITD–positive AML. Patients were randomized 1:1 to quizartinib (n=268) or placebo (n=271) with induction/consolidation and assigned maintenance. The primary endpoint was overall survival (OS). At the primary analysis (minimum 24 months’ follow-up after the last patient was randomized), OS favored quizartinib (HR 0.78; 95% CI 0.62–0.98; 2-sided p=0.0324). CR rates were 55% in both arms; median CR duration was 38.6 months (quizartinib) vs 12.4 months (placebo).
Label note: Quizartinib is not indicated as maintenance monotherapy following allogeneic HSCT; OS improvement has not been demonstrated in that setting.
Safety/program: Boxed warning includes QT prolongation, torsades de pointes, and cardiac arrest; available only via Vanflyta REMS.