Azacitidine · Onureg, Vidaza

What is Azacitidine?

Azacitidine is a pyrimidine nucleoside analog that inhibits DNA and RNA methyltransferases. It is incorporated into DNA and RNA, reducing methylation, altering gene expression, and inducing apoptosis in leukemic cells.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as

Onureg, Vidaza, CC-486, NSC-102816, U-18496

Drug type

Small molecule

Targets

DNA methyltransferase, RNA methyltransferase

Mechanism

Inhibitor of DNA methyltransferase and RNA methyltransferase

Pathways

DNA methylation, RNA methylation, gene expression, apoptosis

Therapy area

Oncology

Developer

Bristol-Myers

In trials of

AAPG, SNDX

FDA status

Approved as Onureg, Vidaza (May 19, 2004), NDA050794 · FDA label

UNII

M801H13NRU

✅ FDA-approved uses

• Refractory anemia (RA) (Vidaza)
• Refractory anemia with ringed sideroblasts (RARS) (Vidaza)
• Refractory anemia with excess blasts (RAEB) (Vidaza)
• Refractory anemia with excess blasts in transformation (RAEB-T) (Vidaza)
• Chronic myelomonocytic leukemia (CMMoL) (Vidaza)
• Juvenile Myelomonocytic Leukemia (JMML) (Vidaza)
• Acute myeloid leukemia in first complete remission or complete remission with incomplete blood count recovery (Onureg)

From the indications section of the FDA label (version of Jan 29, 2024). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Acute Myeloid LeukemiaAMLChronic Myelomonocytic LeukemiaCMMLEpigenetic TherapyHigh-risk Myelodysplastic SyndromeMDSMyelodysplastic SyndromesNon-Small Cell Lung Cancer

Sources

• FDA label (DailyMed)
• FDA label Onureg (2026-06-12), indications
• FDA label Azacitidine (2025-05-26), indications
• FDA label Vidaza (2024-01-29), indications
• NCI Thesaurus
• ClinicalTrials.gov NCT04358393

Updated Oct 3, 2026.