Drug · Small Molecule

Azacitidine

CI-DRUG-00000091Explore in graph →NCIt C288 CHEMBL1489 CIViC Approved

Regulatory

Approvals (31)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

emaRegulatory7 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
EU EMA	—	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.	approved	Jan 5, 2024

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 18 hours agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	192	113	57	514	31	CA, EU, US	May 19, 2004	1999-07
Leukemia	Approved	Phase 4	159	92	50	388	2	EU, US	Sep 1, 2020	2000-05
Non-Hodgkin Lymphoma	Phase3	Phase 3	16	11	3	43	0	—	—	Mar 3, 2006
Hodgkin Lymphoma	Phase3	Phase 3	4	2	1	8	0	—

Curated evidence

Clinical evidence (7)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–7 of 7 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
IDH1 Mutation1
Azacitidine + Ivosidenib

Clinical trials

Trials with this intervention (521)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–50 of 521 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT05554393	Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial)	recruiting	Phase 2	153	National Cancer Institute (NCI)NIH	3	Sep 28, 2026	clinicaltrials
NCT06694454	Neoadjuvant Inhaled Azacytidine With Platinum-Based Chemotherapy and Durvalumab (MEDI4736) - a Combined Epigenetic-Immunotherapy (AZA-AEGEAN) Regimen for Operable Early-Stage Non-Small Cell Lung Cancer (NSCLC)

Molecule

Record

Kind: Small Molecule
Mechanism: DNA (cytosine-5)-methyltransferase 3A inhibitor; DNA (cytosine-5)-methyltransferase 1 inhibitor; DNA inhibitor; RNA inhibitor
Targets: 2 genes
UNII: M801H13NRU
Aliases: 4-Amino-1-beta-D-ribofuranosyl-1,3,5-triazin-2(1H)-one, 5-AC, 5-Azacitidine, 5-Aza-cytidine, 5-Azacytidine, 5 AZC, Azacitidina, Azacitidine, Azacitidine accord, AZACITIDINE AZACITIDINE, Azacitidine betapharm, Azacitidine celgene, Azacitidine kabi, Azacitidine mylan, Azacytidine, Azacytidine, 5-, Ladakamycin, Mylosar, NAT-AZACITIDINE, NSC-102816, Onureg, ONUREG, PMS-AZACITIDINE, REDDY-AZACITIDINE, U-18,496, U-18496, Vidaza, VIDAZA

Data updated 21 hours agoSource updated unknown

Identifiers

Codes (40)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01BC07	AZACITIDINE
DIN (Health Canada)	02336707

IDH1 R1322
Azacitidine + IvosidenibCombination	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	A	Supports Sensitivity Response	5	submitted	EID12899 In the double-blind, placebo-controlled, phase III AGILE trial (NCT03173248), 146 patients with newly diagnosed IDH1-mutated acute myeloid leukemia (AML) who were ineligible for intensive induction ch… (full text at CIViC) PMID 35443108 · Montesinos et al., 2022 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	A	Supports Sensitivity Response	5	submitted	EID12901 This post hoc long-term follow-up analysis of the phase 3, double-blind, placebo-controlled AGILE trial (NCT03173248) reports updated outcomes for 148 patients with newly diagnosed IDH1-mutated AML in… (full text at CIViC) PMID 40706052 · Montesinos et al., 2025 · Open in CIViC	civic