# Azacitidine

Source: https://onco.cc/drugs/azacitidine/  
OnCo record `azacitidine` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A gentle chemotherapy that switches silenced genes back on. With venetoclax it became the standard for older people with AML who cannot take intensive treatment.

## Summary

Approved for MDS (2004) and, in combination with venetoclax, for newly diagnosed AML in patients unfit for intensive chemotherapy (VIALE-A, 2020). Oral azacitidine (Onureg, CC-486) is approved as maintenance after intensive induction (QUAZAR AML-001: OS 24.7 vs 14.8 months). Backbone partner for IDH inhibitors (AGILE), menin inhibitors, and FLT3 inhibitors in trials.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Vidaza / Onureg (oral)
- Modality: Small-molecule hypomethylating agent (cytotoxic)
- Mechanism: Cytidine analogue incorporated into RNA and DNA; traps and depletes DNA methyltransferases, reactivating silenced tumour-suppressor genes and inducing differentiation and apoptosis.
- Approvals: US 2004: Myelodysplastic syndromes; US 2020: Newly diagnosed AML unfit for intensive chemotherapy, with venetoclax (VIALE-A); US 2020: Oral azacitidine (Onureg) maintenance after intensive induction (QUAZAR AML-001)
- Dosing: Subcutaneous or IV (Vidaza); oral (Onureg); 75 mg/m² daily for 7 days of each 28-day cycle (with venetoclax in AML); Onureg 300 mg daily for 14 of 28 days as maintenance
- Toxicity (grade 3+): Neutropenia 42%; Febrile neutropenia 42%

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Azacitidine
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Azacitidine

## Connected records

- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute myeloid leukaemia in older or unfit patients](https://onco.cc/cancers/aml-older-unfit/), [Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia)](https://onco.cc/cancers/advanced-systemic-mastocytosis/), [Chronic myeloid leukaemia, accelerated and blast phase](https://onco.cc/cancers/cml-advanced-phase/), [Chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms](https://onco.cc/cancers/cmml/), [FLT3-mutated acute myeloid leukaemia](https://onco.cc/cancers/aml-flt3/), [Higher-risk myelodysplastic syndromes](https://onco.cc/cancers/mds-higher-risk/), [IDH1- and IDH2-mutated acute myeloid leukaemia](https://onco.cc/cancers/aml-idh/), [Lower-risk myelodysplastic syndromes](https://onco.cc/cancers/mds-lower-risk/), [Myelodysplastic syndromes / neoplasms (MDS)](https://onco.cc/cancers/mds/), [Nodal T-follicular helper cell lymphoma, angioimmunoblastic type (angioimmunoblastic T-cell lymphoma)](https://onco.cc/cancers/angioimmunoblastic-t-cell-lymphoma/), [NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia](https://onco.cc/cancers/aml-npm1-kmt2a/), [Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma)](https://onco.cc/cancers/peripheral-t-cell-lymphoma/), [Secondary and therapy-related acute myeloid leukaemia](https://onco.cc/cancers/aml-secondary/), [Systemic mastocytosis](https://onco.cc/cancers/systemic-mastocytosis/)
- technologies: [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)](https://onco.cc/technologies/epigenetic-drugs/), [Ex vivo drug sensitivity screening in blood cancers (EXALT)](https://onco.cc/technologies/functional-precision-medicine-haematology/)
- companies: [Aptose Biosciences](https://onco.cc/companies/aptose/), [Bristol Myers Squibb](https://onco.cc/companies/bms/), [Dr. Reddy's Laboratories](https://onco.cc/companies/dr-reddys/)
- pathways: [Acute myeloid leukaemia (KEGG map)](https://onco.cc/pathways/aml-signalling/), [Epigenetic reprogramming](https://onco.cc/pathways/epigenetic-reprogramming/), [Intrinsic apoptosis (BCL-2 family)](https://onco.cc/pathways/apoptosis-bcl2/), [Menin / KMT2A (HOXA9-MEIS1 axis)](https://onco.cc/pathways/menin-kmt2a/), [MicroRNAs in cancer](https://onco.cc/pathways/micrornas-in-cancer/)
- trials: [A Clinical Trial of TQB3909 Tablets in Combination With Azacitidine for the Treatment of Myeloid Malignancies](https://onco.cc/trials/nct07011186/), [A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)](https://onco.cc/trials/nct04256317/), [A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)](https://onco.cc/trials/nct04988555/), [A Phase 2 Study of MAX-40279 Combined With Venetoclax and Azacitidine in Unfit Newly Diagnosed Acute Myeloid Leukemia](https://onco.cc/trials/nct07743112/), [A Pivotal Study of APG-2575 (Lisaftoclax) Combined With Azacitidine in the Treatment of Acute Myeloid Leukemia](https://onco.cc/trials/nct06389292/), [A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia](https://onco.cc/trials/nct05883956/), [A Study Evaluating Intensive Chemotherapy With or Without Glasdegib or Azacitidine With or Without Glasdegib In Patients With Previously Untreated Acute Myeloid Leukemia](https://onco.cc/trials/nct03416179/), [A Study of BGB-11417 in Participants With Myeloid Malignancies](https://onco.cc/trials/nct04771130/), [A Study of BL-M11D1 in Combination With Cytarabine + Daunorubicin or Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia](https://onco.cc/trials/nct07255872/), [A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML](https://onco.cc/trials/nct04730258/), [A Study of Cusatuzumab Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia Who Are Not Candidates for Intensive Chemotherapy](https://onco.cc/trials/nct04023526/), [A Study of Gilteritinib, Venetoclax and Azacitidine as a Combined Treatment for People Newly Diagnosed With Acute Myeloid Leukemia](https://onco.cc/trials/nct05520567/), [A Study of Tagraxofusp in Combination With Venetoclax and Azacitidine in Adults With Untreated CD123+ Acute Myeloid Leukemia Who Cannot Undergo Intensive Chemotherapy](https://onco.cc/trials/nct06456463/), [A Study to Assess Adverse Events and Change in Disease Activity When Intravenous (IV) Pivekimab Sunirine is Given in Combination With Oral Venetoclax and IV or Subcutaneous Azacitidine in Adult Participants With Acute Myeloid Leukemia (AML)](https://onco.cc/trials/nct07581002/), [A Study to Assess Safety, Tolerability and Preliminary Efficacy of Bexmarilimab in Combination With Standard of Care in Patients With Hematological Malignancies](https://onco.cc/trials/nct05428969/), [A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy](https://onco.cc/trials/nct02677922/), [A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Japanese Participants With Acute Myeloid Leukemia (AML) in Complete Remission](https://onco.cc/trials/nct05197426/), [A Trial of AK117 (Anti-CD47 Antibody) in Patients With Acute Myeloid Leukemia](https://onco.cc/trials/nct04980885/), [AGILE](https://onco.cc/trials/agile/), [An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.](https://onco.cc/trials/nct05907057/), [AZA-001](https://onco.cc/trials/aza-001/), [Beat AML Master Trial](https://onco.cc/trials/beat-aml-master-trial/), [BMT CTN 1102](https://onco.cc/trials/bmt-ctn-1102/), [Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia](https://onco.cc/trials/nct03850574/), [Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMML](https://onco.cc/trials/nct04842604/), [FCN-338 in Combination With Azacitidine or Chemotherapy in Myeloid Neoplasms](https://onco.cc/trials/nct06858618/), [Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2](https://onco.cc/trials/nct04229979/), [IMGN632 as Monotherapy or With Venetoclax and/or Azacitidine for Participants With CD123-Positive Acute Myeloid Leukemia](https://onco.cc/trials/nct04086264/), [Magrolimab + Azacitidine Versus Azacitidine + Placebo in Untreated Participants With Myelodysplastic Syndrome (MDS)](https://onco.cc/trials/nct04313881/), [PBSS1113 in Combination With Azacitidine to Treat Patients With AML/MDS](https://onco.cc/trials/nct07761533/), [Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML)](https://onco.cc/trials/nct03268954/), [QUAZAR AML-001](https://onco.cc/trials/quazar-aml-001/), [Randomized Phase IIB Trial of Oral Azacytidine Plus Romidepsin Versus Investigator's Choice in PTCL](https://onco.cc/trials/nct04747236/), [Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab.](https://onco.cc/trials/nct05201066/), [Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML](https://onco.cc/trials/nct07007312/), [Study of Azacitidine，Venetoclax，and Flumatinib in Newly Diagnosed Ph-positive Acute Leukemia and CML-AP/BP Patients](https://onco.cc/trials/nct05433532/), [Study of Lisaftoclax (APG-2575) Single Agent and Combination With Therapy in Patients Relapsed/Refractory AML](https://onco.cc/trials/nct04501120/), [Study of Magrolimab in Combination With Azacitidine Versus Physician's Choice of Venetoclax in Combination With Azacitidine or Intensive Chemotherapy in Patients With TP53 Mutant Acute Myeloid Leukemia That Have Not Been Treated](https://onco.cc/trials/nct04778397/), [Study of Magrolimab Versus Placebo in Combination With Venetoclax and Azacitidine in Participants With Acute Myeloid Leukemia](https://onco.cc/trials/nct05079230/), [Study of MP0533 in Patients Acute Myeloid Leukemia or Myelodysplastic Syndrome](https://onco.cc/trials/nct05673057/), [Study of SLS009 (Formerly GFH009) a Potent Highly Selective CDK9 Inhibitor in Patients With Hematologic Malignancies and High-Risk Newly Diagnosed AML](https://onco.cc/trials/nct04588922/), [Study to Compare Azacitidine Plus Pevonedistat Versus Azacitidine in Patients With Acute Myeloid Leukemia Not Eligible for Standard Chemotherapy](https://onco.cc/trials/nct04090736/), [Study to Evaluate CCS1477 (Inobrodib) in Haematological Malignancies](https://onco.cc/trials/nct04068597/), [Study to Evaluate the Safety and Tolerability of EP0042](https://onco.cc/trials/nct04581512/), [VERONA](https://onco.cc/trials/verona/), [VIALE-A](https://onco.cc/trials/viale-a/)
- key papers: [AGILE: ivosidenib plus azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy](https://onco.cc/key-papers/paper-agile-ivosidenib-azacitidine-nejm-2022/), [AZA-001: azacitidine versus conventional care in higher-risk myelodysplastic syndromes](https://onco.cc/key-papers/paper-aza-001-fenaux-lancet-oncol-2009/), [AZA-AML-001: azacitidine versus conventional care in older patients with acute myeloid leukaemia and more than 30 percent blasts](https://onco.cc/key-papers/paper-aza-aml-001-dombret-blood-2015/), [QUAZAR AML-001: oral azacitidine maintenance for acute myeloid leukaemia in first remission](https://onco.cc/key-papers/paper-quazar-aml-001-wei-nejm-2020/), [VIALE-A: venetoclax plus azacitidine for older adults with acute myeloid leukaemia who cannot have intensive chemotherapy](https://onco.cc/key-papers/paper-viale-a-venetoclax-azacitidine-nejm-2020/)
- pairings: [Menin inhibitor + venetoclax + azacitidine](https://onco.cc/pairings/menin-plus-venetoclax-hma/), [Venetoclax + hypomethylating agent](https://onco.cc/pairings/venetoclax-plus-hma/)
- ideas: [Keep dormant cells asleep instead of trying to kill them](https://onco.cc/ideas/idea-bio2-pro-dormancy-therapy/), [Shorter venetoclax courses in unfit AML](https://onco.cc/ideas/idea-shortened-venetoclax/), [Unmask hidden antigens with a short epigenetic course before immunotherapy](https://onco.cc/ideas/idea-bio2-epigenetic-priming-cold-tumours/)
- biomarkers: [RHOA G17V](https://onco.cc/biomarkers/rhoa-g17v/)
- targets: [DNA methyltransferase 3A (DNMT3A)](https://onco.cc/targets/dnmt3a/), [DNMT1 (DNA methyltransferase 1)](https://onco.cc/targets/dnmt1/)
- people: [Stephen B. Baylin](https://onco.cc/people/stephen-baylin/)
- terms: [7+3 induction chemotherapy](https://onco.cc/terms/seven-plus-three/), [AML with myelodysplasia-related gene mutations (AML-MR)](https://onco.cc/terms/aml-myelodysplasia-related/), [Epigenetic progenitor theory: cancer without a first mutation](https://onco.cc/terms/epigenetic-progenitor-theory/), [Hypomethylating agents (azacitidine, decitabine)](https://onco.cc/terms/hma/), [IPSS-R and IPSS-M (myelodysplastic syndrome risk scores)](https://onco.cc/terms/ipss-m-ipss-r/)
- institutions: [USC Norris Comprehensive Cancer Center](https://onco.cc/institutions/usc-norris/)
- drugs: [Decitabine](https://onco.cc/drugs/decitabine/), [Lenzilumab](https://onco.cc/drugs/lenzilumab/), [Pevonedistat](https://onco.cc/drugs/pevonedistat/)
- roadmaps: [Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome](https://onco.cc/roadmaps/epigenetics-roadmap/)

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