Data updated: May 26, 2026
VIDAZA (azacitidine)
Nucleic Acid Synthesis Inhibitors
Trial Activity: Stable 146 active trials
Oncology
Approved 2004-05-19
VIDAZA 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; Juvenile Myelomonocytic Leukemia.
How VIDAZA Works
Vidaza is a pyrimidine nucleoside analog of cytidine that exerts antineoplastic effects through DNA hypomethylation and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. By inhibiting DNA methyltransferase, it may restore normal function to genes critical for cellular differentiation and proliferation. Its cytotoxic effects specifically target rapidly dividing cells; notably, non-proliferating cells are relatively insensitive to azacitidine.
Development Insights
M.D. Anderson Cancer Center conducting 46 trials (12%)
371 indications explored (Broad Platform)
→ acute myeloid leukemia (118 trials)
→ myelodysplastic syndrome (52 trials)
→ myelodysplastic syndromes (50 trials)
4
Indications
--
Phase 3 Trials
3
Priority Reviews
22
Years on Market
Details
- Status
- Prescription
- First Approved
- 2004-05-19
- Patent Cliff
- 2029
- Routes
- INTRAVENOUS, SUBCUTANEOUS
- Dosage Forms
- POWDER
VIDAZA Approval History
2005
2006
2007
2008
2009
2010
2011
2012
2013
2014
2015
2016
2017
2018
2019
2020
2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
20 FDA actions from 2004 to 2024 · 3 indication expansions
Jan 2017 SUPPL
Mfg
Feb 2015 SUPPL Priority
Mfg
May 2013 SUPPL Priority
Mfg
What VIDAZA Treats
7 indicationsVIDAZA is approved for 7 conditions since its original approval in 2004. 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
- Juvenile Myelomonocytic Leukemia
Source: FDA Label
VIDAZA Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
5
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
10
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
10
Same indication, different mechanism — what else might this patient receive?
Unlock 19 more competitors across all three rings.
Upgrade to Pro 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 VIDAZA's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications VIDAZA treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to VIDAZA
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
AZACITIDINE
AZACITIDINE
PHARMOBEDIENT
Shared indications:
Myelodysplastic SyndromeRefractory AnemiaRefractory Anemia with Ringed Sideroblasts +3 more
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Clinical Trial Registry
371 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 371 trials
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Pro Active Pipeline
Ongoing clinical trials by development phase
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Pro Key Completed Trials
Completed studies with published results, ranked by significance
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Trial Timeline
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.
VIDAZA FDA Label Details
Indications & Usage
FDA Label (PDF)VIDAZA 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; Juvenile Myelomonocytic Leukemia.
VIDAZA Patents & Exclusivity
Exclusivity: May 2029
Exclusivity
ODE-399
Until May 2029
Source: FDA Orange Book
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment