Azacitidine

Nucleoside Metabolic Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Azacitidine is an ofp drug in the Nucleoside Metabolic Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2004. Its mechanism of action is classified as Nucleic Acid Synthesis Inhibitors. DrugCentral lists 2 molecular targets, including DNMT1 and DNMT3A. Recorded indications include Acute myeloid leukemia, disease, Chronic myelomonocytic leukemia and Myelodysplastic syndrome.

• Approval status: ofp (first approved 2004 by the FDA).
• Drug class: Nucleoside Metabolic Inhibitor.
• Mechanism of action: Nucleic Acid Synthesis Inhibitors.
• Molecular targets: DNMT1 and DNMT3A (2 listed in DrugCentral).
• Indications: Acute myeloid leukemia, disease, Chronic myelomonocytic leukemia and Myelodysplastic syndrome.
• ATC code: L01BC07 (Pyrimidine analogues).
• Molecular formula: C8H12N4O5; molecular weight 244.21 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (3)

• Acute myeloid leukemia, disease
• Chronic myelomonocytic leukemia
• Myelodysplastic syndrome

Contraindications (7)

• Advanced Malignant Neoplasm of Liver
• Breastfeeding (mother)
• Hepatic failure
• Kidney disease
• Neutropenic disorder
• Pregnancy, function
• Thrombocytopenic disorder

Mechanism & targets

• Mechanism of action (MoA): Nucleic Acid Synthesis Inhibitors
• Established pharmacologic class: Nucleoside Metabolic Inhibitor

Molecular targets (2)

Target	Gene	Action	Activity
DNA (cytosine-5)-methyltransferase 1	DNMT1	inhibitor	IC50 6.52
DNA (cytosine-5)-methyltransferase 3A	DNMT3A	inhibitor	—

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2019-08-02	EMA	—	Celgene Europe BV
2011-01-21	PMDA	—	Nippon Shinyaku Co., Ltd.	Yes
2004-05-19	FDA	—	CELGENE	Yes

ATC classification

ATC code	Therapeutic hierarchy
L01BC07	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › ANTINEOPLASTIC AGENTS › ANTIMETABOLITES › Pyrimidine analogues

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: KRAS, NRAS, PTPN11
• FDA label: CBL, KRAS, NRAS, PTPN11
• HCSC label

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Febrile neutropenia	5178.6
Myelosuppression	3266.1
Acute myeloid leukaemia	2420.5
Death	2316.1
Acute myeloid leukaemia recurrent	2056.3
Neutropenia	1972.5
Thrombocytopenia	1476.6
Sepsis	1166.8
Cytopenia	1160.4
Pneumonia	1115.2
Pancytopenia	1087.2
Neutrophil count decreased	950.8

Chemical structure

Full physicochemical, ADMET and 3D-structure analysis of Azacitidine is on its molecule page.

• Molecular formula: C₈H₁₂N₄O₅
• Molecular weight: 244.21 g/mol
• CAS number: —
• InChIKey: NMUSYJAQQFHJEW-KVTDHHQDSA-N
• PubChem CID: 9444

NC1=NC(=O)N(C=N1)[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Azacitidine used for?

DrugCentral lists the following indications for Azacitidine: Acute myeloid leukemia, disease, Chronic myelomonocytic leukemia and Myelodysplastic syndrome.

How does Azacitidine work?

Azacitidine is classified as Nucleic Acid Synthesis Inhibitors; its molecular targets in DrugCentral include DNMT1 (inhibitor) and DNMT3A (inhibitor).

When was Azacitidine first approved?

Azacitidine was first approved by the FDA in 2004.

What drug class does Azacitidine belong to?

Azacitidine is in the Nucleoside Metabolic Inhibitor class.

What is the chemical formula of Azacitidine?

Azacitidine has the molecular formula C8H12N4O5 and a molecular weight of 244.21 g/mol.

Sources

• DrugCentral drug card 25 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL1489 — compound record and bioactivity
• PubChem CID 9444 — chemical identifiers
• DrugBank DB00928 — drug record
• FDA UNII M801H13NRU — substance identity
• DailyMed: FDA labels for Azacitidine — current prescribing information (label search)
• UniProt P26358 (DNMT1) — target protein
• UniProt Q9Y6K1 (DNMT3A) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.