Back to discover
Drug

Azacitidine

Approved · FDA / EMA
Emerging researchLate-stage developmentCooling momentum
Also known as 4-amino-1-beta-D-ribofuranosyl-s-triazin-2(1H)-one, 5 azacytidine, 5-Azacitidine, 5-azacytidine+6 more

4-amino-1-beta-D-ribofuranosyl-s-triazin-2(1H)-one, 5 azacytidine, 5-Azacitidine, 5-azacytidine, Onureg, Vidaza, Ladakamycin, Azacitidina, Azacytidine, Azacitidinum.

RxNorm1251ChEMBLCHEMBL1489UNIIM801H13NRU
8
Research papers
74
Active clinical trials
12
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Randomized Phase II Study Comparing Cytarabine + Daunorubicin (7 + 3) vs (Daunorubicin and Cytarabine) Liposome, Cytarabine + Daunorubicin + Venetoclax, Azacitidine + Venetoclax, and (Daunorubicin and Cytarabine) Liposome + Venetoclax in Patients Aged 59 or Younger Who Are Considered High-Risk (Adverse) Acute Myeloid Leukemia As Determined by MYELOMATCH; A MYELOMATCH SubStudy

Clinical trial2026-07-31Results expected Q1 2027 · ClinicalTrials.gov

A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

Clinical trial2026-07-29Results expected Q3 2027 · ClinicalTrials.gov

A Randomized Phase II Study of Venetoclax and HMA-Based Therapies for the Treatment of Older and Unfit Adults With Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia (AML): A MyeloMATCH Treatment Trial

Clinical trial2026-07-10Results expected Q2 2029 · ClinicalTrials.gov

A Phase II/III Randomized Study of R-MiniCHOP With or Without CC-486 (Oral Azacitidine) in Participants Age 75 Years or Older With Newly Diagnosed Diffuse Large B Cell Lymphoma, Grade IIIB Follicular Lymphoma, Transformed Lymphoma, and High-Grade B-Cell Lymphomas With MYC AND BCL2 and/or BCL6 Rearrangements

Clinical trial2026-07-08Results expected Q1 2027 · ClinicalTrials.gov

Label change: AZACITIDINE (ANDA204949)

Regulatory2026-06-09FDA

Label change: AZACITIDINE (ANDA204949)

Regulatory2026-06-09FDA

Label change: AZACITIDINE (ANDA204949)

Regulatory2026-06-09FDA

Approval: Azacitidine Kabi (EMA)

Regulatory2024-01-05EMA

Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML.

Research2018-01-18Blood

Epigenetic induction of adaptive immune response in multiple myeloma: sequential azacitidine and lenalidomide generate cancer testis antigen-specific cellular immunity.

Research2012-07-23British journal of haematology

Continued azacitidine therapy beyond time of first response improves quality of response in patients with higher-risk myelodysplastic syndromes.

Research2011-01-10Cancer

Effects of azacitidine compared with conventional care regimens in elderly (≥ 75 years) patients with higher-risk myelodysplastic syndromes.

Research2010-05-06Critical reviews in oncology/hematology

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Azacitidine
Aliases & brands
4-amino-1-beta-D-ribofuranosyl-s-triazin-2(1H)-one5 azacytidine5-Azacitidine5-azacytidineOnuregVidazaLadakamycinAzacitidinaAzacytidineAzacitidinum
RxNorm CUI
1251
ChEMBL ID
CHEMBL1489
UNII
M801H13NRU
Regulatory jurisdictions
emafda

Regulatory timeline

12 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2008-12-17
Latest approval
2024-01-05
Authorities
FDA · EMA
Total events
12
fdaU.S. Food and Drug Administration· 6 events
2026-06-09Label change
Label change: AZACITIDINE (ANDA204949)
Evidence ↗
2026-06-09Label change
Label change: AZACITIDINE (ANDA204949)
Evidence ↗
2026-06-09Label change
Label change: AZACITIDINE (ANDA204949)
Evidence ↗
emaEuropean Medicines Agency· 6 events
2024-01-05Approval
Approval: Azacitidine Kabi (EMA)
Indication: Azacitidine Kabi is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation(HSCT) with: Intermediate-2 and high-risk… Show full indication

Azacitidine Kabi is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation(HSCT) with: Intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the international prognostic scoring system (IPSS), Chronic myelomonocytic leukaemia (CMML) with 10-29% marrow blasts without myeloproliferative disorder, Acute myeloid leukaemia (AML) with 20-30% blasts and multi-lineage dysplasia, according to World Health Organization (WHO) classification, AML with > 30% marrow blasts according to the WHO classification.

Evidence ↗
2021-06-17Approval
Approval: Onureg (EMA)
Indication: Onureg is indicated as maintenance therapy in adult patients with acute myeloid leukaemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood… Show full indication

Onureg is indicated as maintenance therapy in adult patients with acute myeloid leukaemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT).

Evidence ↗
2020-03-27Approval
Approval: Azacitidine Mylan (EMA)
Indication: Azacitidine Mylan is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) with: intermediate 2 and high risk… Show full indication

Azacitidine Mylan is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) with: intermediate 2 and high risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS), chronic myelomonocytic leukaemia (CMML) with 10 29% marrow blasts without myeloproliferative disorder, acute myeloid leukaemia (AML) with 20 30% blasts and multi lineage dysplasia, according to World Health Organisation (WHO) classification, AML with > 30% marrow blasts according to the WHO classification.

Evidence ↗

Data from the U.S. Food and Drug Administration (U.S. Food and Drug Administration), public domain (CC0).

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

189 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
189
registered trials across all phases
LATEST COMPLETION 2025
74
Active studies
53
Recruiting
30
Late-stage (III+)
57
Completed
58
Discontinued
PHASE DISTRIBUTIONn = 189
Early Phase 14Phase 147Phase 1 / 245Phase 263Phase 2 / 34Phase 319Phase 44Phase N / A3

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

REMAIN1: Relapse Prevention With Maintenance Oral Azacitidine in Transplant Eligible Patients With Acute Myeloid Leukemia Not Proceeding to Transplant Due to Racial or Socioeconomic Disparities

RecruitingPH IVVirginia Commonwealth Universityn=122029

A Phase 4 Study of Venetoclax in Combination With Azacitidine in Newly Diagnosed Subjects With Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy in China

Active, not recruitingPH IVAbbVien=442026

The Efficacy and Safety of Azacytidine Combined With HAG Regimen Versus Azacytidine for Elderly Patients With Newly Diagnosed Myeloid Malignancy: a Prospective, Randomized Controlled Trial

Unknown statusPH IVShenzhen Second People's Hospitaln=1142023

A Phase 4, Open-Label, Single-Arm Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Azacitidine in Adult Taiwanese Subjects With Higher-Risk Myelodysplastic Syndromes.

CompletedPH IVCelgenen=442013

A Randomized, Open-labelled, Multicenter Trial Evaluating Efficacy and Safety of A Reduced Venetoclax Exposure To Seven Days Versus Standard Continuous Venetoclax Exposure Combined With Azacitidine in Treatment Naïve Subjects With Acute Myeloid Leukemia Who Are Ineligible for Intensive Induction

Not yet recruitingPH IIIGustave Roussy, Cancer Campus, Grand Parisn=2622031

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 1 Open-label, Multi-center Study of the Safety, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS)

CompletedPH IPureTechn=1012025

A Phase 3 Open-Label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation

CompletedPH IIIAstellas Pharma Global Development, Inc.n=3712025

A Phase I/IB Pilot Study of CC-486 Combined With Lenalidomide and Obinutuzumab for Relapsed/Refractory Indolent B-Cell Lymphoma

CompletedPH IJoseph Tuscanon=82025

A Multicenter, Phase I/II Study of Sequential Epigenetic and Immune Targeting in Combination With Nab-Paclitaxel/Gemcitabine in Patients With Advanced Pancreatic Ductal Adenocarcinoma.

CompletedPH I/IIGWT-TUD GmbHn=752024

A Randomized Phase II/III Trial of "Novel Therapeutics" Versus Azacitidine in Newly Diagnosed Patients With Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS), Age 60 or Older LEAP: Less-Intense AML Platform Trial

CompletedPH II/IIINational Cancer Institute (NCI)n=762024

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Compare Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia in Complete Remission

CompletedPH IIICelgenen=4722024

Research activity

8 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20092018
Most influential

Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study.

The Lancet. Oncology · 2009 · 1,943 cites

Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2010 · 701 cites

Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML.

Blood · 2018 · 166 cites

Continued azacitidine therapy beyond time of first response improves quality of response in patients with higher-risk myelodysplastic syndromes.

Cancer · 2011 · 148 cites

Management and supportive care measures for adverse events in patients with myelodysplastic syndromes treated with azacitidine*.

European journal of haematology · 2010 · 92 cites

Effects of azacitidine compared with conventional care regimens in elderly (≥ 75 years) patients with higher-risk myelodysplastic syndromes.

Critical reviews in oncology/hematology · 2010 · 84 cites
Recent papers

Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML.

Blood · 2018 · 166 cites

Epigenetic induction of adaptive immune response in multiple myeloma: sequential azacitidine and lenalidomide generate cancer testis antigen-specific cellular immunity.

British journal of haematology · 2012 · 36 cites

Continued azacitidine therapy beyond time of first response improves quality of response in patients with higher-risk myelodysplastic syndromes.

Cancer · 2011 · 148 cites

Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2010 · 701 cites

Management and supportive care measures for adverse events in patients with myelodysplastic syndromes treated with azacitidine*.

European journal of haematology · 2010 · 92 cites

Effects of azacitidine compared with conventional care regimens in elderly (≥ 75 years) patients with higher-risk myelodysplastic syndromes.

Critical reviews in oncology/hematology · 2010 · 84 cites
Major research themes2
Lymphocyte Transfusion1Peripheral Blood Stem Cell Transplantation1
Journals, researchers & institutions
Top journals
  • British journal of haematology2
  • Blood1
  • Cancer1
  • Critical reviews in oncology/hematology1
  • European journal of haematology1
  • Journal of clinical oncology : official journal of the American Society of Clinical Oncology1
Leading researchers
  • Fenaux P6
  • Mufti GJ6
  • Seymour JF6
  • Backstrom J5
  • Gore SD5
  • Hellström-Lindberg E5
  • Santini V5
  • Silverman LR5
Leading institutions
free-text, unnormalised
  • Assistance Publique-Hôpitaux de Paris and Paris 13 University1
  • Azienda Ospedaliera Universitaria Careggi1
  • Colorado Blood Cancer Institute1
  • Department of Pathology and Laboratory Medicine1
  • Hospital Avicenne1
  • Lineberger Comprehensive Cancer Center1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.