Last update 12 Jan 2026

Azacitidine

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
4-amino-1-beta-D-ribofuranosyl-s-triazin-2(1H)-one, 5-azacytidine, 5-AZC
+ [20]
Target
Action
inhibitors
Mechanism
DNMT1 inhibitors(DNA (cytosine-5)-methyltransferase 1 inhibitors), DNA methylation inhibitors, Epigenetic drug
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (19 May 2004),
RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Accelerated assessment (European Union), Priority Review (China)
Login to view timeline

Structure/Sequence

Molecular FormulaC8H12N4O5
InChIKeyNMUSYJAQQFHJEW-KVTDHHQDSA-N
CAS Registry320-67-2

External Link

R&D Status

Approved
10 top approved records.
Login
to view more data
IndicationCountry/LocationOrganizationDate
Juvenile Myelomonocytic Leukemia
United States
20 May 2022
Myeloproliferative Disorders
European Union
24 Mar 2020
Myeloproliferative Disorders
Iceland
24 Mar 2020
Myeloproliferative Disorders
Liechtenstein
24 Mar 2020
Myeloproliferative Disorders
Norway
24 Mar 2020
Philadelphia chromosome positive chronic myelogenous leukemia
China
24 Apr 2017
High Risk Myelodysplastic Syndrome
Australia
30 Nov 2009
Acute Myeloid Leukemia
European Union
17 Dec 2008
Acute Myeloid Leukemia
Iceland
17 Dec 2008
Acute Myeloid Leukemia
Liechtenstein
17 Dec 2008
Acute Myeloid Leukemia
Norway
17 Dec 2008
Chronic Myelomonocytic Leukemia
European Union
17 Dec 2008
Chronic Myelomonocytic Leukemia
Iceland
17 Dec 2008
Chronic Myelomonocytic Leukemia
Liechtenstein
17 Dec 2008
Chronic Myelomonocytic Leukemia
Norway
17 Dec 2008
Myelodysplastic Syndromes
United States
19 May 2004
Developing
10 top R&D records.
Login
to view more data
IndicationHighest PhaseCountry/LocationOrganizationDate
Immunoblastic LymphadenopathyPhase 3
Japan
06 Nov 2018
ThrombocytopeniaPhase 3
United States
26 Apr 2013
ThrombocytopeniaPhase 3
Australia
26 Apr 2013
ThrombocytopeniaPhase 3
Belgium
26 Apr 2013
ThrombocytopeniaPhase 3
Brazil
26 Apr 2013
ThrombocytopeniaPhase 3
Canada
26 Apr 2013
ThrombocytopeniaPhase 3
Czechia
26 Apr 2013
ThrombocytopeniaPhase 3
Denmark
26 Apr 2013
ThrombocytopeniaPhase 3
Finland
26 Apr 2013
ThrombocytopeniaPhase 3
France
26 Apr 2013
Login to view more data

Clinical Result

Indication
Phase
Evaluation
View All Results
Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
57
(Arm A: Acute Myeloid Leukemia (AML))
azooxaodfj = dukogqqmar zylxaxizxh (pjrqyqybdb, phnekjfcqm - mctlndsnvv)
-
10 Dec 2025
(Arm B: High risk Myleodysplastic Syndrome (MDS))
azooxaodfj = brfehoykkc zylxaxizxh (pjrqyqybdb, owoxfshqky - rdsvtdqqyq)
Not Applicable
668
Intensive Chemotherapy
ohiaaawkvo(jyicszkrhi) = fqtggpyogq rzvzwopqam (nmffetkkzm )
Positive
06 Dec 2025
AZA+VEN
ohiaaawkvo(jyicszkrhi) = fxqpfcprff rzvzwopqam (nmffetkkzm )
Not Applicable
-
150
EPO
zzigvdjqgj(pkltqwulpu) = 5 patients ndadiadnix (hiykhmwptt )
Positive
06 Dec 2025
Phase 1/2
Acute Myeloid Leukemia
First line
FLT3mut+
44
dbvoemugjr(uofnprnpfz) = abevkfzsca dzimhzrhgp (yeonwzgrrd, 46 - 88)
Positive
06 Dec 2025
dbvoemugjr(uofnprnpfz) = ykgutqnbba dzimhzrhgp (yeonwzgrrd, 43 - 84)
Not Applicable
37
XAB regimen
ipmjyljrph(upqsikbjqc) = htqszgjwux opdwmcvkie (opfloswnmy )
Positive
06 Dec 2025
XAB regimen
ipmjyljrph(upqsikbjqc) = cjhifthsxi opdwmcvkie (opfloswnmy )
Phase 2
23
kccdsdaayg(nskghqlrbs) = yxigeqxmtl fdhystnpku (bbrgaxsqck )
Positive
06 Dec 2025
Not Applicable
48
(FLT3-ITD mutated AML + Refractory/Relapsed disease or persistent MRD)
hfnskqfoca(vzdeypyfoz) = lvgilzaapn uxicpwykms (xvedmdxzuf, 3.8 - 21.0)
Positive
06 Dec 2025
Not Applicable
321
azacufqftf(zaocydpcey) = pcpytmniaw fndvbfuiid (aowjiurhus )
Positive
06 Dec 2025
azacufqftf(zaocydpcey) = gtysakiaht fndvbfuiid (aowjiurhus )
Phase 1
Acute Myeloid Leukemia
First line
Bcl-2 expression
20
vaacawpbaj(mxjxaorixm) = BH3 profiling predicted VEN resistance as early as day 5 of treatment. In the overall cohort, a significant increase in sensitivity to Mcl-1 (MS-1) (p < 0.01) and Bcl-xL (HRK) (p < 0.05) peptides was observed at day 5 post-treatment. Among responders, no significant changes were detected, whereas in non-responders, a significant increase in sensitivity to the Mcl-1 (MS-1) peptide was observed at day 5 post-treatment (p < 0.01) yorsjsebly (enyoaammak )
Positive
06 Dec 2025
Phase 2
140
oral-Aza+Vididencel
rnxrxfexmx(xasbshbdst) = mboziicdoa kuzdbrdnjy (hutcfntqik )
Positive
06 Dec 2025
rnxrxfexmx(xasbshbdst) = zoqrzaebhh kuzdbrdnjy (hutcfntqik )
Login to view more data

Translational Medicine

Boost your research with our translational medicine data.
Boost your research with our translational medicine data.

Deal

Boost your decision using our deal data.
Boost your decision using our deal data.

Core Patent

Boost your research with our Core Patent data.
Boost your research with our Core Patent data.

Clinical Trial

Identify the latest clinical trials across global registries.
Identify the latest clinical trials across global registries.

Approval

Accelerate your research with the latest regulatory approval information.
Accelerate your research with the latest regulatory approval information.

Regulation

Understand key drug designations in just a few clicks with Synapse.
Understand key drug designations in just a few clicks with Synapse.
AI Agents Built for Biopharma Breakthroughs
Accelerate discovery. Empower decisions. Transform outcomes.
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.
Bio
Bio Sequences Search & Analysis
Sign up for free
Chemical
Chemical Structures Search & Analysis
Sign up for free