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midostaurin + cytarabine + daunorubicin + gemtuzumab ozogamicin

Development stage
Unknown
Lead developer
Novartis
Modality
DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics, Covalent Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules
Administration
Oral, Intravenous
01

Overview

This is a four-drug combination regimen used primarily for the treatment of acute myeloid leukaemia (AML), especially in patients with FLT3 mutations and/or CD33-positive disease. The regimen includes: - **Midostaurin**: A small molecule tyrosine kinase inhibitor that blocks multiple kinases, including FLT3, thereby inhibiting cell growth and proliferation. - **Cytarabine**: A chemotherapy agent that interferes with DNA synthesis, leading to cell death in rapidly dividing cells. - **Daunorubicin**: An anthracycline chemotherapy drug that intercalates into DNA and inhibits topoisomerase II, causing DNA damage and apoptosis. - **Gemtuzumab ozogamicin**: An antibody-drug conjugate consisting of a monoclonal antibody targeting CD33 linked to the cytotoxic agent calicheamicin. It binds to CD33 on leukemia cells, is internalized, and releases calicheamicin to induce cell death. This combination is typically used as induction therapy for newly diagnosed AML patients who are fit for intensive therapy. Gemtuzumab ozogamicin specifically targets CD33-positive leukemic blasts; midostaurin is added when there are FLT3 mutations[1][2][5].

Brand names
RydaptMylotarg
Other names
GODA
02

Targets

PRKCI (Protein kinase C iota)CD33 (Myeloid cell surface antigen CD33)KIT (c-KIT proto-oncogene receptor tyrosine kinase)PDGFRA (Platelet-derived growth factor receptor alpha)DNA polymerase familyCDK1 (Cyclin-dependent kinase 1)TOP2A (DNA topoisomerase II)SYK (Spleen Tyrosine Kinase)FLT3 (Fms related receptor tyrosine kinase 3)

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