What kind of drug does Midostaurin belong to?

Midostaurin (Midostaurin) is a targeted anticancer drug that is a treatment for acute myeloid leukemia (AML). Midostaurin can be used to treat AML if a patient's cancer has a change (mutation) in the FLT3 gene; it can be used alone as maintenance therapy, or it can be used with chemotherapy drugs, and doctors will check for the mutation before starting the drug.

1. Acute myeloid leukemia (AML): It can be used in combination with cytarabine and daunorubicin as a component of induction therapy, and then in combination with high-dose cytarabine as a component of combined therapy for newly diagnosed AML containing Flt-3 mutations (designated as an orphan drug). However, midostaurin is not suitable for single-agent induction therapy in patients with AML.

In preclinical studies, midostaurin was found to be active against the oncogenic CD135 (FMS-like tyrosine kinase 3 receptor, FLT3). Clinical trials mainly focus on relapsed/refractory AML and MDS, including single drug and combination drug studies. Following a successful Phase II clinical trial, midostaurin was found to extend survival in patients with FLT3-mutated AML when used in combination with conventional induction and consolidation therapy in a randomized Phase III clinical trial.

1. Systemic mastocytosis (SM): Midostaurin is indicated as a monotherapy for the treatment of aggressive systemic mastocytosis (ASM), systemic mastocytosis with hematologic malignancy (SM-AHN; also known as systemic mastocytosis with associated hematological non-mast cell lineage disease [SM-AHNMD]), and mast cell leukemia (designated as an orphan drug).

More than95% of adult patients with systemic mastocytosis and approximately 40% of children with cutaneous mastocytosis are positive for the D816V c-Kit activating mutation, which renders c-Kit resistant to currently available tyrosine kinase inhibitors. Midostaurin is an investigational treatment for patients with advanced systemic mastocytosis and D816V c-Kit mutations, and some patients have achieved clinical efficacy. Midostaurin was effective in patients with advanced systemic mastocytosis, including a highly lethal variant of mast cell leukemia, in an open-label study of patients with mastocytosis-related organ damage.

Midostaurin is a tyrosine kinase inhibitor (TKI) cancer growth retardant. Tyrosine kinase inhibitors block chemical messengers (enzymes) called tyrosine kinases. Tyrosine kinases help send growth signals in cells, so blocking them can prevent cells from growing and dividing. Midostaurin is a capsule preparation. Patients need to take the drug appropriately under the guidance of a doctor. They can take the drug once a day in the morning and evening. Regardless of whether the stomach is full or empty, it may affect the amount of the drug entering the blood.