Main indications and application scope of Quizartinib

Quizartinib is a novel FLT3 kinase inhibitor indicated for the treatment of adult patients with acute myeloid leukemia (AML) harboring FLT3 internal tandem duplication mutations (FLT3-ITD). Such mutations account for approximately 25% to 30% of AML and are closely related to high disease recurrence rates and poor prognosis. Quizartinib can effectively prevent the differentiation, stagnation and proliferation of tumor cells by highly selectively inhibiting the abnormal activation of the FLT3 signaling pathway, thereby achieving precise inhibition of leukemia cells. It is one of the important advances in the current molecular targeted therapy of AML. The drug is usually used in combination with cytarabine and anthracyclines during the induction and consolidation phases of treatment, and is subsequently used as a single agent for long-term maintenance treatment to reduce the risk of relapse.

Quizatinib is unique in that it is a second-generation selective FLT3 inhibitor. Its mechanism of action has been improved for the resistance mechanism of FLT3-ITD mutations. Compared with the first-generation drugs, it has higher target binding stability and fewer non-target side effects. In clinical guidelines in European and American countries, quizartinib has been recommended as one of the first-line standard treatment options for newly diagnosed FLT3-positive AML, especially for patients who carry mutations when first diagnosed. In addition to standard indications, some studies have shown that quizartinib also has potential efficacy in patients with relapsed or refractory AML and can be used as a transitional treatment option to improve patients' chances of receiving hematopoietic stem cell transplantation (HSCT).

Currently, the application of quizartinib in the context of precision medicine is constantly expanding. The scientific community is exploring its potential in combination therapy with other targeted drugs such as the BCL-2 inhibitor venetoclax to further extend recurrence-free survival.

Overall, quizartinib not only represents a major breakthrough in the AML treatment model, but also marks the in-depth development of "genotyping-driven therapy" in the field of hematological tumors, providing a more precise and personalized treatment path for patients with FLT3 mutations.

Reference materials:https://go.drugbank.com/drugs/DB12874