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.

openfdaRegulatory8 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	Efficacy supplement 2026-04-09 (see label)	approved	Apr 9, 2026	openfda
US FDA	—	Efficacy supplement 2022-05-20 (see label)	approved	May 20, 2022

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 17 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 301–350 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
NCT04912063	Study to Evaluate Adverse Events and Movement of Lemzoparlimab in Body When Used Intravenously (IV) With Azacitidine Subcutaneously or IV and Venetoclax Orally in Participants With Acute Myeloid Leukemia and With Azacitidine With or Without Venetoclax in Participants With Myelodysplastic Syndrome	terminated	Phase 1	40	AbbVieINDUSTRY	7	Feb 26, 2024	clinicaltrials
NCT01812252	Chemotherapy in Treating Patients With Myelodysplastic Syndrome Before Donor Stem Cell Transplant

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 20 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