Rydapt (midostaurin)

Description

What is Rydapt (midostaurin) for?

Rydapt (midostaurin) is indicated for the treatment of patients with^[1,3]:

• Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation positive as detected by an FDA-approved test, in combination with chemotherapy (standard cytarabine and daunorubicin induction and cytarabine consolidation). Rydapt (midostaurin)is not indicated as a single-agent induction therapy for the treatment of patients with AML.
• Aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

How does Rydapt (midostaurin) work?

The active substance in Rydapt is midostaurin, a kinase inhibitor that works by blocking several enzymes that promote cell growth^[2,4].

Midostaurin demonstrated the ability to inhibit FLT3 receptor signalling and cell proliferation, and it induced apoptosis in leukemic cells expressing ITD and TKD mutant FLT3 receptors or overexpressing wild type FLT3 and PDGF receptors. Midostaurin also demonstrated the ability to inhibit KIT signalling, cell proliferation, and histamine release and induce apoptosis in mast cells^[1,4].

Is Rydapt (midostaurin) approved?

Rydapt (midostaurin) was approved for patients newly diagnosed acute myeloid leukemia (AML) who have a specific genetic mutation called FLT3, in combination with chemotherapy; and for patients with systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL)^[1,3,5,6,7] by:

• Food and Drug Administration (FDA) (USA) on April 28, 2017
• European Medicines Agency (EMA) (EU) on October 25, 2017
• Therapeutic Goods Administration (TGA) (Australia) on May 17, 2018
• Health Canada on July 21, 2017
• Medsafe (New Zealand) on Oct 24, 2019

How do I take Rydapt (midostaurin)?

The standard dosage for AML is:

• 50 mg (2 capsules) orally twice daily with food.

The standard dosage for ASM, SM-AHN, and MCL is:

• 100 mg (4 capsules) orally twice daily with food.

Complete information about Rydapt (midostaurin) dosage and administration can be found in the references section.

Note: Consult your treating doctor for personalised dosing.

Are there any known side effects of Rydapt (midostaurin)?

Among the common side effects in patients with AML are low levels of white blood cells with fever (febrile neutropenia), nausea and inflammation of the mucous membranes (mucositis)^[1].

Among the patients with ASM, SM-AHN, or MCL the most common adverse reactions (≥ 20%) were nausea, vomiting, diarrhea, edema, musculoskeletal and abdominal pain^[1].