Last reviewed · How we verify
5-Azacitidine
Inhibits DNA methyltransferase
Inhibits DNA methyltransferase Used for Acute myeloid leukemia.
At a glance
| Generic name | 5-Azacitidine |
|---|---|
| Also known as | VIDAZA, Azacitidine, 5-azacytidine, 5-aza, Vidaza |
| Sponsor | Shirley Ryan AbilityLab |
| Drug class | Hypomethylating agent |
| Target | DNA methyltransferase |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 2 |
Mechanism of action
5-Azacitidine is a hypomethylating agent that works by inhibiting DNA methyltransferase enzymes, leading to a decrease in DNA methylation and an increase in gene expression.
Approved indications
- Acute myeloid leukemia
Common side effects
- Myelosuppression
- Fatigue
- Nausea
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:
- 5-Azacitidine CI brief — competitive landscape report
- 5-Azacitidine updates RSS · CI watch RSS
- Shirley Ryan AbilityLab portfolio CI