How it is administered

Midostaurin is taken by mouth as a soft capsule. Each capsule contains 25 mg of midostaurin. The medication should be taken with food, twice daily, at approximately 12-hour intervals. The dose for most blood cancers is 100 mg (four 25 mg capsules) twice daily. It is important not to open or crush the capsules. If a dose is missed or vomited, do not make up the dose; take the next dose at the usual scheduled time.

How it works

Midostaurin is a type of medicine known as a kinase inhibitor. It works by blocking the activity of certain proteins called receptor tyrosine kinases, which are involved in the growth and survival of cancer cells. Specifically, midostaurin targets proteins such as FLT3, KIT, and PDGFR, which can be abnormally active in some blood cancers. By blocking these proteins, midostaurin helps to stop cancer cells from growing and can trigger them to die (a process called apoptosis). This action can slow the progression of the disease and help control symptoms.

Midostaurin is especially useful in cancers where these proteins are mutated or overactive, such as some forms of acute myeloid leukemia (AML) and advanced systemic mastocytosis. Its ability to target multiple kinases makes it a valuable option for patients whose cancer cells rely on these pathways for survival.

Who Should take it

Midostaurin is approved for adults with certain blood cancers:

• Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive, in combination with standard chemotherapy (cytarabine and daunorubicin).
• Aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

It is not approved as a single-agent induction therapy for AML. Patients should have their cancer tested for the FLT3 mutation before starting midostaurin for AML. For advanced systemic mastocytosis and related conditions, midostaurin can be used as a single agent. Your healthcare provider will decide if midostaurin is appropriate based on your specific diagnosis and mutation status.

Who should not take it

You should not take midostaurin if you have a known allergy or hypersensitivity to midostaurin or any of its ingredients. Signs of a serious allergic reaction may include anaphylactic shock, difficulty breathing, flushing, chest pain, or swelling of the airways or tongue.

Midostaurin may cause harm to unborn babies, so it should not be used during pregnancy. Women of childbearing potential should use effective contraception during treatment and for 4 months after the last dose. Men with female partners of childbearing potential should also use contraception during treatment and for 4 months after the last dose. Breastfeeding is not recommended during treatment and for 4 months after the last dose.

Commonly used with

For acute myeloid leukemia (AML), midostaurin is commonly used in combination with standard chemotherapy drugs such as cytarabine and daunorubicin during induction and consolidation phases. It may also be used with supportive medications like anti-emetics to help manage nausea and vomiting.

For advanced systemic mastocytosis and related conditions, midostaurin is typically used as a single agent, but supportive care medications may also be prescribed.

Commonly tested with

In clinical studies, midostaurin has been tested in combination with standard chemotherapy agents, particularly cytarabine and daunorubicin, for AML. It has also been studied as a single agent in patients with advanced systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, and mast cell leukemia.

Drug interaction studies have evaluated midostaurin with strong CYP3A4 inhibitors (such as ketoconazole, itraconazole) and inducers (such as rifampicin), as well as with drugs metabolized by CYP2B6, BCRP, and OATP1B1.