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AZACITIDINE
AZACITIDINE is a Nucleoside Metabolic Inhibitor [EPC] drug. It is currently FDA-approved (first approved 2004) for Refractory Anemia (RA), Refractory Anemia with Ringed Sideroblasts (RARS), Refractory Anemia with Excess Blasts (RAEB).
Azacitidine is used to treat various types of cancer, including Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, and Hodgkin Lymphoma, as well as previously untreated Peripheral T-cell Lymphoma. It is administered as CC-486, which is a form of azacitidine.
At a glance
| Generic name | AZACITIDINE |
|---|---|
| Drug class | Nucleoside Metabolic Inhibitor [EPC] |
| Modality | Small molecule |
| Phase | FDA-approved |
| First approval | 2004 |
Approved indications
- Refractory Anemia (RA)
- Refractory Anemia with Ringed Sideroblasts (RARS)
- Refractory Anemia with Excess Blasts (RAEB)
- Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
- Chronic Myelomonocytic Leukemia (CMMoL)
Common side effects
- Nausea
- Vomiting
- Diarrhea
- Fatigue/asthenia
- Constipation
- Upper respiratory tract infection
- Pneumonia
- Abdominal pain
- Arthralgia
- Decreased appetite
- Febrile neutropenia
- Dizziness
Serious adverse events
- Pneumonia
- Febrile neutropenia
- Sepsis
- Nausea
- Diarrhea
- Vomiting
Key clinical trials
- Testing the Combination of an Anti-Cancer Drug, Iadademstat, With Other Anti-Cancer Drugs (Venetoclax and Azacitidine) for Treating AML (PHASE1)
- The Safety and Tolerability of LBS-007 in Patients With Relapsed or Resistant Acute Leukaemias (PHASE1, PHASE2)
- 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)
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 |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| 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 CI brief — competitive landscape report
- AZACITIDINE updates RSS · CI watch RSS
Frequently asked questions about AZACITIDINE
What is AZACITIDINE?
What is AZACITIDINE used for?
What drug class is AZACITIDINE in?
When was AZACITIDINE approved?
What development phase is AZACITIDINE in?
What are the side effects of AZACITIDINE?
Related
- Drug class: All Nucleoside Metabolic Inhibitor [EPC] drugs
- Indication: Drugs for Refractory Anemia (RA)
- Indication: Drugs for Refractory Anemia with Ringed Sideroblasts (RARS)
- Indication: Drugs for Refractory Anemia with Excess Blasts (RAEB)
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing