How it is administered

Gilteritinib is taken by mouth as a tablet. Each tablet contains 40 mg of gilteritinib (as the free base). The recommended dose is 120 mg (usually 3 tablets) taken once daily, with or without food. Tablets should be swallowed whole with water and should not be broken, crushed, or chewed.

How it works

Gilteritinib is a type of targeted therapy known as a kinase inhibitor. It works by blocking the activity of a protein called FLT3 (FMS-like tyrosine kinase 3), which is often mutated and overactive in some cases of acute myeloid leukemia (AML). By inhibiting FLT3 and other related kinases, gilteritinib helps to stop the growth and survival of leukemia cells that rely on these signals.

This targeted approach is especially useful for patients whose AML has a specific FLT3 mutation, as these mutations can make the cancer more aggressive and harder to treat with standard chemotherapy. By blocking the abnormal signals, gilteritinib can slow down or stop the progression of the disease, and in some cases, help achieve remission.

Who Should take it

Gilteritinib is indicated for adults with acute myeloid leukemia (AML) that has relapsed (come back) or is refractory (not responding to previous treatment), and who have a FLT3 mutation. The presence of the FLT3 mutation must be confirmed by an FDA-approved test before starting treatment.

It is generally used when other treatments have not worked or the disease has returned, and is specifically targeted for those whose leukemia cells have the FLT3 mutation, as these patients are more likely to benefit from this medication.

Who should not take it

Gilteritinib should not be taken by anyone who has had a severe allergic reaction (hypersensitivity) to gilteritinib or any of its ingredients. Signs of a severe allergic reaction can include rash, itching, swelling, severe dizziness, or trouble breathing.

It should also be avoided in women who are pregnant or may become pregnant, as it can cause harm to an unborn baby. Women should use effective contraception during treatment and for 6 months after the last dose, and men with female partners should use contraception during treatment and for 4 months after the last dose. Breastfeeding is not recommended during treatment and for 2 months after the last dose.

Commonly used with

Gilteritinib is often used alone for the treatment of relapsed or refractory AML with FLT3 mutations. In clinical trials, it was compared to various chemotherapy regimens, but it is not typically combined with other chemotherapy drugs as standard practice for this indication.

However, patients may receive supportive medications such as corticosteroids if differentiation syndrome develops, or other treatments to manage side effects.

Commonly tested with

In clinical studies, gilteritinib was compared to high-intensity chemotherapy regimens (such as MEC: mitoxantrone, etoposide, cytarabine; and FLAG-IDA: fludarabine, cytarabine, idarubicin, G-CSF) and low-intensity regimens (such as low-dose cytarabine or azacitidine). It is also tested in combination with supportive care medications, including corticosteroids for differentiation syndrome and other drugs to manage side effects.