On 21 November 2018, the US Food and Drug Administration (FDA) accepted a New Drug Application (NDA) and granted priority review to quizartinib, an oral, highly potent and selective FMS-like tyrosine kinase 3 (FLT3) inhibitor, for the treatment of adult patients with relapsed/refractory (R/R) FLT3-ITD-mutated acute myeloid leukemia (AML). This comes after the FDA granted a Breakthrough Therapy Designation to quizartinib for the treatment of R/R FLT3-ITD AML in August 2018.
The NDA application for quizartinib was based on data from the pivotal phase III randomized QuANTUM-R study, which is assessing the efficacy and safety of quizartinib (60 mg, with a 30 mg lead-in for 15 days) versus salvage chemotherapy (SC) in patients with R/R FLT3-ITD-mutated AML. Data from this study were presented by Jorge Cortes from the MD Anderson Cancer Center, Houston, Texas, at the 23rd Congress of the European Hematology Association, Stockholm, Sweden in June 2018. Single-agent quizartinib significantly prolonged overall survival in patients with R/R FLT3-ITD AML compared to SC. More results from this phase III randomized study are reported here.
Jorge Cortes, in an interview with the AML Global Portal (AGP), highlighted that the QuANTUM-R trial was a “very positive study”, and he hopes that it leads to a “regulatory approval” for quizartinib as it “offers an advantage” over the current treatment options for these difficult to treat patients. The drug manufacturers also added that “quizartinib has the potential to meaningfully advance treatment for patients with relapsed or refractory FLT3-ITD AML” if approved.