Azacitidine is a pyrimidine nucleoside analog of cytidine. Azacitidine is 4-amino-1-β-D-ribofuranosyl-s-triazin-2(1H)-one.The empirical formula is C8H12N4O5. The molecular weight is 244. Azacitidine is a white to off-white solid.

Azacitidine, also known as 5-aza-2′-deoxycytidine, sold under the brand name Vidaza among others, was first synthesized in Czechoslovakia as potential chemotherapeutic agents for cancer.

As a chemical analog of cytidine, a nucleoside in DNA and RNA, Azacitidine is a medication used for the treating myelodysplastic syndrome, myeloid leukemia, and juvenile myelomonocytic leukemia.

Azacitidine is indicated for treatment of patients with the following myelodysplastic syndrome subtypes: refractory anemia or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia.

Azacitidine is believed to exert its antineoplastic effects by causing hypomethylation of DNA and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. The concentration of Azacitidine required for maximum inhibition of DNA methylation in vitro does not cause major suppression of DNA synthesis. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The cytotoxic effects of Azacitidine cause the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. Non-proliferating cells are relatively insensitive to Azacitidine.

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