Drug intelligence / Profile preview

panobinostat + idarubicin + cytarabine

Development stage
Unknown
Lead developer
Novartis
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

This is a **combination regimen of panobinostat, idarubicin, and cytarabine** investigated for the treatment of high-risk or newly diagnosed acute myeloid leukemia (AML), especially in younger and elderly patient populations. **Panobinostat** is a histone deacetylase inhibitor (HDACi) that modulates gene expression and DNA repair, thereby sensitizing leukemic cells to DNA-damaging chemotherapy. **Idarubicin** is an anthracycline antineoplastic agent that functions as a topoisomerase II inhibitor, leading to DNA breaks and apoptosis. **Cytarabine** (also known as ara-C) is an antimetabolite that inhibits DNA synthesis. Preclinical and clinical evidence supports a mechanistic synergy: panobinostat suppresses BRCA1, CHK1, and RAD51, reducing DNA repair and enhancing cytarabine/idarubicin-induced cell death and checkpoint abrogation. This regimen showed promising tolerability and efficacy, with reported complete remission rates of 43–64% and manageable adverse events in high-risk AML patients[1][2][3][5].

Other names
panobinostat + idarubicin + ara-C
02

Targets

DNA polymerase familyHDAC (HDAC family)TOP2A (DNA topoisomerase II)

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