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Azacitidine (AZA)
Azacitidine (AZA) is a DNA methyltransferase inhibitor Small molecule drug developed by Memorial Sloan Kettering Cancer Center. It is currently FDA-approved for Myelodysplastic syndromes (MDS), Acute myeloid leukemia (AML), Chronic myelomonocytic leukemia (CMML). Also known as: Vidaza, Azadine, 5-Azacytidine, 5-AZA.
Azacitidine inhibits DNA methyltransferase, leading to DNA demethylation and reactivation of silenced tumor suppressor genes.
Azacitidine inhibits DNA methyltransferase, leading to DNA demethylation and reactivation of silenced tumor suppressor genes. Used for Myelodysplastic syndromes (MDS), Acute myeloid leukemia (AML), Chronic myelomonocytic leukemia (CMML).
At a glance
| Generic name | Azacitidine (AZA) |
|---|---|
| Also known as | Vidaza, Azadine, 5-Azacytidine, 5-AZA, 5-AZC |
| Sponsor | Memorial Sloan Kettering Cancer Center |
| Drug class | DNA methyltransferase inhibitor |
| Target | DNA methyltransferase (DNMT) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Azacitidine is a nucleoside analog that incorporates into DNA and inhibits DNA methyltransferase (DNMT), reducing aberrant DNA methylation in cancer cells. This demethylation reactivates epigenetically silenced tumor suppressor genes and can induce differentiation and apoptosis in myelodysplastic and leukemic cells. It is thought to restore normal gene expression patterns disrupted by hypermethylation in hematologic malignancies.
Approved indications
- Myelodysplastic syndromes (MDS)
- Acute myeloid leukemia (AML)
- Chronic myelomonocytic leukemia (CMML)
Common side effects
- Myelosuppression (anemia, thrombocytopenia, neutropenia)
- Nausea and vomiting
- Fatigue
- Fever
- Injection site reactions
- Constipation
Key clinical trials
- Testing the Combination of an Anti-Cancer Drug, Iadademstat, With Other Anti-Cancer Drugs (Venetoclax and Azacitidine) for Treating AML (PHASE1)
- 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 (PHASE2)
- Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia (PHASE2)
- Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial) (PHASE2)
- Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial) (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia (PHASE1)
- Azacitidine and Abatacept in Relapsed or Refractory T-Cell Lymphoma (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Azacitidine (AZA) CI brief — competitive landscape report
- Azacitidine (AZA) updates RSS · CI watch RSS
- Memorial Sloan Kettering Cancer Center portfolio CI
Frequently asked questions about Azacitidine (AZA)
What is Azacitidine (AZA)?
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What is Azacitidine (AZA) used for?
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Is Azacitidine (AZA) also known as anything else?
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Related
- Drug class: All DNA methyltransferase inhibitor drugs
- Target: All drugs targeting DNA methyltransferase (DNMT)
- Manufacturer: Memorial Sloan Kettering Cancer Center — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Myelodysplastic syndromes (MDS)
- Indication: Drugs for Acute myeloid leukemia (AML)
- Indication: Drugs for Chronic myelomonocytic leukemia (CMML)
- Also known as: Vidaza, Azadine, 5-Azacytidine, 5-AZA, 5-AZC
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing