Common brand names

• Rydapt

How it is administered

Midostaurin is taken by mouth as a soft capsule, usually with food. For systemic mastocytosis, the recommended dose is 100 mg (four 25 mg capsules) twice daily. Capsules should be swallowed whole and not opened or crushed. If a dose is missed or vomited, do not take an extra dose; just take the next dose at the usual scheduled time.

How it works

Midostaurin is a type of medicine called a kinase inhibitor. It works by blocking the action of certain abnormal proteins (kinases) in the body that help cancer cells grow and survive. In systemic mastocytosis, midostaurin targets the KIT protein, including the D816V mutant form commonly found in this disease. By blocking this protein, midostaurin helps slow down or stop the growth and activity of abnormal mast cells.

Midostaurin also affects other proteins involved in blood cancers, such as FLT3, PDGFR, and members of the PKC family. This broad activity helps it work against several types of blood cancers, including aggressive forms of systemic mastocytosis and certain types of acute myeloid leukemia (AML).

Who should take it

Midostaurin is used to treat adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL). It is also used in combination with chemotherapy for adults with newly diagnosed FLT3-mutated acute myeloid leukemia (AML).

For systemic mastocytosis, midostaurin is appropriate for patients who have advanced forms of the disease, especially when symptoms are severe or the disease is progressing. Your doctor will determine if midostaurin is right for you based on your specific diagnosis and overall health.

Who should not take it

You should not take midostaurin if you are allergic to midostaurin or any of its ingredients. Signs of a serious allergic reaction can include anaphylactic shock, swelling of the airways or tongue, chest pain, or difficulty breathing.

Midostaurin can cause harm to an unborn baby, so it should not be used during pregnancy. Women who are pregnant or may become pregnant should not take this medication. It is also not recommended for women who are breastfeeding, as it is not known if the drug passes into breast milk. The safety and effectiveness of midostaurin in children have not been established.

Commonly used with

For AML, midostaurin is used in combination with standard chemotherapy drugs such as cytarabine and daunorubicin. For systemic mastocytosis, it is typically used as a single agent, but your doctor may prescribe supportive medications such as anti-nausea drugs to help manage side effects.

Commonly tested with

Midostaurin has been tested with standard chemotherapy regimens in AML (cytarabine and daunorubicin). In systemic mastocytosis, it has been studied as a single agent. Sometimes, supportive medications like antiemetics (for nausea) are used alongside midostaurin to help with side effects.