Midostaurin is the first generation FLT3-targeting drug

Midostaurin is the first generation FLT3 targeted drug. It is the first molecular targeted drug developed and approved for the treatment of FLT3 mutant acute myeloid leukemia (AML). It was developed by Novartis and was approved by the US FDA in 2017 for use in combination with chemotherapy to treat patients with newly diagnosed FLT3 mutation-positive AML. The launch of midostaurin marks the beginning of the era of targeted therapy for AML, providing new treatment ideas for this highly heterogeneous disease.

As a first-generation FLT3 inhibitor, midostaurin has a broad mechanism of action and can not only inhibit FLT3 (including ITD and TKD mutation type), can also act on a variety of other tyrosine kinases, such as KIT, PDGFR, VEGFR, etc. Therefore, its targets are relatively "miscellaneous" and its relative selectivity is poor, which also affects its efficacy and tolerability to a certain extent. However, when used in combination with conventional chemotherapy, midostaurin can still significantly prolong the overall survival of patients and is recommended by multiple international guidelines as an important part of initial treatment.

Although midostaurin opened the door toFLT3 targeted therapy, with the deepening of research, scientists have successively developed second and third generations of FLT3 with higher selectivity and lower drug resistance. Inhibitors, such as Quizartinib (Quizartinib) and Gilitinib (Gilteritinib). Compared with midostaurin, these subsequent drugs are optimized in terms of selectivity, efficacy, and response to drug-resistant mutations, and are particularly suitable for patients with relapsed/refractory AML or those who are not sensitive to first-generation drugs.

In general, midostaurin is the foundational drug in the field of FLT3 targeted therapy. It is a first-generation FLT3 inhibitor. Although it is not as good as subsequent drugs in selectivity and resistance management, it is very useful in new diagnosesFLT3mutationsAML still have important clinical value in patients. Its combination with chemotherapy is still one of the standard treatment strategies recommended by most guidelines, which has brought survival benefits to AML patients and laid a solid foundation for the subsequent development of FLT3-targeted drugs.

Reference materials:https://medlineplus.gov/druginfo/meds/a617033.html