TheraRadar
Data updated: May 26, 2026

AZACITIDINE

Nucleic Acid Synthesis Inhibitors
Oncology Approved 2013-09-16

AZACITIDINE is indicated for the treatment of Myelodysplastic Syndrome; Refractory Anemia; Refractory Anemia with Ringed Sideroblasts; Refractory Anemia with Excess Blasts; Refractory Anemia with Excess Blasts in Transformation; Chronic Myelomonocytic Leukemia.

Source: FDA Label • PHARMOBEDIENT • Nucleoside Metabolic Inhibitor
Jump to: Indications Approvals Trials Competitors Similar Safety Patents

How AZACITIDINE Works

Azacitidine is a pyrimidine nucleoside analog of cytidine. It is believed to exert antineoplastic

Source: FDA Label
16
Indications
--
Phase 3 Trials
12
Years on Market

Details

Status
Discontinued
First Approved
2013-09-16
Routes
INTRAVENOUS, SUBCUTANEOUS
Dosage Forms
POWDER, TABLET

Companies

PHARMOBEDIENT HETERO LABS LTD VI AMNEAL EUGIA PHARMA Teva SHILPA MEDICARE NATCO PHARMA LTD Lupin MEITHEAL RELIANCE LIFE JIANGSU HANSOH PHARM Dr. Reddy's Cipla ACCORD HLTHCARE EUROHLTH INTL SARL MSN LABS PVT LTD
Active Ingredient: AZACITIDINE

AZACITIDINE Approval History

2014
2015
2016
2017
2018
2019
2020
2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
49 FDA actions from 2013 to 2025
Jul 2025 SUPPL
Label · Labeling
May 2025 ORIGINAL
Update
Apr 2025 SUPPL
Label · Labeling

What AZACITIDINE Treats

6 indications

AZACITIDINE is approved for 6 conditions since its original approval in 2013. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Myelodysplastic Syndrome
  • Refractory Anemia
  • Refractory Anemia with Ringed Sideroblasts
  • Refractory Anemia with Excess Blasts
  • Refractory Anemia with Excess Blasts in Transformation
  • Chronic Myelomonocytic Leukemia
Source: FDA Label

AZACITIDINE Competitive Set

Pro

Three rings of competition based on shared molecular targets and treated indications.

Direct competitors

5

Same target(s) AND same indication — head-to-head.

FRINDOVYX
AVYXA HOLDINGS
2023 13 indic.
DOXORUBICIN HYDROCHLORIDE (LIPOSOMAL)
Baxter
2013 10 indic.

MoA expansion candidates

10

Same target(s), different indications — where else is this mechanism being explored?

LOMUSTINE
CARNEGIE
2025 2 indic.
KYXATA
AVYXA HOLDINGS
2025 1 indic.

Indication competitors

10

Same indication, different mechanism — what else might this patient receive?

GRAFAPEX
MEDEXUS
2025 2 indic.
IMKELDI
SHORLA ONCOLOGY
2024 7 indic.
Unlock 19 more competitors across all three rings.
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Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.

What's emerging in AZACITIDINE's indications

See all emerging drugs →

Phase 3 candidates targeting molecules with no FDA-approved drug, in indications AZACITIDINE treats. First-in-class if their pivotal trials read out positive.

Ceralasertib AZD6738 ~ Mixed — Ph3 readout
AstraZeneca
ATR · small-molecule ATR kinase inhibitor · 2 Phase 3 trials

Drugs Similar to AZACITIDINE

3 of 4

FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.

VIDAZA
AZACITIDINE
6 shared
Bristol-Myers Squibb
Shared indications:
Myelodysplastic SyndromeRefractory AnemiaRefractory Anemia with Ringed Sideroblasts +3 more
DECITABINE
DECITABINE
4 shared
Novartis
Shared indications:
Refractory AnemiaRefractory Anemia with Ringed SideroblastsRefractory Anemia with Excess Blasts +1 more
INQOVI
CEDAZURIDINE
4 shared
TAIHO ONCOLOGY
Shared indications:
Refractory AnemiaRefractory Anemia with Ringed SideroblastsRefractory Anemia with Excess Blasts +1 more
View all 4 similar drugs Pro
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Clinical Trial Registry

367 trials
Trial Sponsor ID Phase Status Title
NCT06514261 NCI-2024-04616 NCI-2024-04616, 24-115 Ph 1 suspended Testing the Combination of an Anti-Cancer Drug, Iadademstat, With Other Anti-Cancer Drugs (Venetoclax and Azacitidine) for Treating AML
NCT06635681 DAV-AML-2024 IIT2024074 Ph 2 recruiting Efficacy of Venetoclax Combined With Intensive Chemotherapy in Different Subgroups of AML
NCT06744556 IIT2024095 Ph 2 active not recruiting HAV Versus DAV/IAV Induction Regimen in Elderly Patients With AML
NCT05401097 OSU-21330 NCI-2022-01324, R01CA262496 Ph 2 recruiting IDH Targeted/Non- Targeted vs Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)
NCT05564390 NCI-2022-07006 NCI-2022-07006, MYELOMATCH Ph 2 recruiting MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
NCT04687761 VEN-A-QUI Ph 1, Ph 2 completed Clinical Trial to Assess the Safety and Tolerability of the Combination of Low-dose Cytarabine or Azacitidine Plus Venetoclax and Quizartinib in Newly Diagnosed AML Patients Aged Equal or More Than 60 Years Old
NCT06317649 NCI-2024-01987 NCI-2024-01987, MM1OA-EA02 Ph 2 recruiting Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial)
NCT05554393 NCI-2022-07534 NCI-2022-07534, AL.6 Ph 2 recruiting Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial)
NCT05554406 NCI-2022-07535 NCI-2022-07535, MM1YA-S01 Ph 2 recruiting Testing the Effects of Novel Therapeutics for Newly Diagnosed, Untreated Patients With High-Risk Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)
NCT04801797 21-113 Ph 2 active not recruiting Venetoclax + Azacitidine vs. Induction Chemotherapy in AML
NCT07581002 REVIVAL M26-092 2025-523724-47-00 Ph 2, Ph 3 not yet recruiting A Study to Assess Adverse Events and Change in Disease Activity When Intravenous (IV) Pivekimab Sunirine is Given in Combination With Oral Venetoclax and IV or Subcutaneous Azacitidine in Adult Participants With Acute Myeloid Leukemia (AML)
NCT04150887 ELEVATE ELEV20235 2019-002808-41, ELEV20235 Ph 1 active not recruiting Cusatuzumab in Combination With Background Therapy for the Treatment of Participants With Acute Myeloid Leukemia
NCT07044687 M23-826 Ph 4 recruiting Study to Assess Adverse Events and Change in Disease Activity of Oral Venetoclax in Combination With Subcutaneous (SC) or Intravenous (IV) Azacitidine in Newly Diagnosed Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Standard Induction Therapy in India
NCT06465953 PyramIDH S095031-178 2023-510155-37 Ph 3 recruiting Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation
NCT06384261 OV-AML-1231 2024-510991-19-00 Ph 2 active not recruiting A Study Comparing Venetoclax and Azacitidine Plus Cusatuzumab to Venetoclax and Azacitidine in Newly Diagnosed AML Ineligible for Intensive Therapy
NCT07388563 10002363 002363-C Ph 1 suspended Azacitidine and Abatacept in Relapsed or Refractory T-Cell Lymphoma
NCT05907057 DIM-95031-006 2022-501709-11 Ph 3 recruiting An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.
NCT05155709 CHDM201I12201 2021-001165-21 Ph 1 completed A Study of Siremadlin in Combination With Venetoclax Plus Azacitidine in Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Chemotherapy.
NCT04401748 Verona M15-954 2023-507153-16-00 Ph 3 active not recruiting Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome
NCT05829226 LYT-200-2022-02 Ph 1 completed A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS)
NCT04493138 2019-1178 NCI-2020-05261, 2019-1178 Ph 1, Ph 2 active not recruiting Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations
NCT06177067 RAVAML NCI-2023-10509 Ph 1 recruiting Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia
NCT05182957 R/R PTCL 01 Ph 2 recruiting Clinical Study on Anti-PD-1 Plus Lenalidomide and Azacitidine in Relapsed/Refractory Peripheral T-cell Lymphoma
NCT04588922 GFH009X2101 Ph 1, Ph 2 recruiting Study of SLS009 (Formerly GFH009) a Potent Highly Selective CDK9 Inhibitor in Patients With Hematologic Malignancies and High-Risk Newly Diagnosed AML
NCT00658814 results posted NCI-2009-00790 NCI-2009-00790, SWOG-S0703 Ph 2 active not recruiting Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia
NCT01522976 results posted NCI-2012-00242 NCI-2012-00242, CDR0000723909 Ph 2 active not recruiting Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
NCT04256317 ASTX030-01 2024-515098-93 Ph 2, Ph 3 recruiting A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
NCT06497062 BGB-11417-2001-IIT Ph 2 active not recruiting Phase II Study of Sonrotoclax Combined With Chemotherapy in the Treatment of Newly Diagnosed Acute Myeloid Leukemia
NCT04501120 APG2575AC101 Ph 1, Ph 2 recruiting Study of Lisaftoclax (APG-2575) Single Agent and Combination With Therapy in Patients Relapsed/Refractory AML
NCT05183035 ITCC-101/APAL2020D 2021-003212-11, 2023-510160-12-00 Ph 3 recruiting Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)
NCT05586074 HEC73543-AML-301 Ph 3 recruiting HEC73543 Versus Salvage Chemotherapy in R/R FLT3-ITD AML
NCT07548983 STUDY00028015 NCI-2025-09157, STUDY00028015 Ph 1 not yet recruiting Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation
NCT03164057 AML16 NCI-2017-00928 Ph 2 active not recruiting A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT05428969 BEXMAB FP2CLI004 2021-002104-12 Ph 1, Ph 2 active not recruiting A Study to Assess Safety, Tolerability and Preliminary Efficacy of Bexmarilimab in Combination With Standard of Care in Patients With Hematological Malignancies
NCT04435691 results posted 2020-0027 NCI-2020-04163, 2020-0027 Ph 1, Ph 2 terminated Magrolimab, Azacitidine, and Venetoclax for the Treatment of Acute Myeloid Leukemia
NCT04771130 BGB-11417-103 2021-003285-12, 2023-508881-14-00 Ph 1, Ph 2 recruiting A Study of BGB-11417 in Participants With Myeloid Malignancies
NCT05955261 AML23 NCI-2023-04138 Ph 2 suspended A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia
NCT07320235 IMAGINE STUDY-25-01212 PRMC-25-108 Ph 1 recruiting Imetelstat Combinations in Relapsed AML
NCT07539818 IIT2026028 Ph 3 not yet recruiting Azacitidine + Venetoclax VS Azacitidineas Maintenance Therapy in AML
NCT03586609 2018-0020 NCI-2018-01318, 2018-0020 Ph 2 recruiting Venetoclax, Cladribine, Low Dose Cytarabine, and Azacitidine in Treating Patients With Previously Untreated Acute Myeloid Leukemia
NCT04140487 2019-0366 NCI-2019-04959, 2019-0366 Ph 1, Ph 2 recruiting Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm
NCT06782542 OLUVENAZA 20240763 Ph 2 recruiting Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction Chemotherapy
NCT05849662 T2020-004 Ph 1, Ph 2 recruiting A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
NCT06656494 ICP-CL-01205 Ph 1 recruiting ICP-248 in Combination With Azacitidine for the Treatment in Patients With Myeloid Malignancies
NCT07469046 SHSYXY-202508-VAH-VA Ph 3 not yet recruiting VAH vs VA in Newly Diagnosed Elderly AML
NCT05554419 NCI-2022-07799 NCI-2022-07799, MM2YA-EA01 Ph 2 not yet recruiting Testing the Use of Combination Therapy in Patients With Persistent Low Level Acute Myeloid Leukemia Following Initial Treatment, The ERASE Study (A MyeloMATCH Treatment Trial)
NCT04284787 results posted NCI-2020-01016 NCI-2020-01016, 2000031641 Ph 2 active not recruiting BLAST MRD AML-2: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 2- A Randomized Phase 2 Study of Anti-PD-1 Pembrolizumab in Combination With Azacitidine and Venetoclax as Frontline Therapy in Unfit Patients With Acute Myeloid Leukemia
NCT06504459 STUDY00026216 NCI-2024-05084, STUDY00026216 Ph 2 recruiting Venetoclax in Combination With Cladribine and Cytarabine Alternating With Azacitidine Plus Venetoclax for the Treatment of Newly Diagnosed Monocytic AML and Active Signaling Mutated AML
NCT07177079 202506290 Ph 1 recruiting High-dose Ascorbate (HDA) in Combination With Standard of Care Azacitidine and Venetoclax in Acute Myeloid Leukemia (AML)
NCT05442216 TAGALONG HCRN AML20-472 Ph 2 recruiting Tagraxofusp and Azacitidine With Venetoclax in Newly Diagnosed Secondary AML After Hypomethylating Agents

Showing 50 of 367 trials

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Active Pipeline

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Ongoing clinical trials by development phase

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

ClinicalTrials.gov ↗ Pro

Full development history with FDA approval milestones

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

AZACITIDINE FDA Label Details

Indications & Usage

FDA Label (PDF)

AZACITIDINE is indicated for the treatment of Myelodysplastic Syndrome; Refractory Anemia; Refractory Anemia with Ringed Sideroblasts; Refractory Anemia with Excess Blasts; Refractory Anemia with Excess Blasts in Transformation; Chronic Myelomonocytic Leukemia.

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Data Sources

FDA Approval: ANDA204949 → Label: DailyMed → Patents: FDA Orange Book → Trials: ClinicalTrials.gov →

Data sourced from official FDA and NIH databases. Click links to verify on original sources.