Azacitidine

CAS 320-67-2

About the API

Systematic name 4-amino-1-β-D-ribofuranosyl-1,3,5-triazin-2(1H)-one
Trade name(s) Vidaza
Molecular Formula C8H12N4O5
Molecular Weight 244.20 g/mol
Physical properties White to off- white powder
Therapeutic category Oncology
Available formulations Injectables
Regulations EU DMF Flag EU DMF US DMF Flag US DMF

General Information

Azacitidine is used to treat myelodysplastic syndromes (MDS), a number of disorders in which not enough healthy blood cells are produced in the bone marrow, leaving patients with low blood counts. In healthy bone marrow, stem cells become red blood cells, white blood cells or platelets. In MDS, this process is dysfunctional. In a significant portion of patients, MDS progresses to a rapidly growing cancer of bone marrow cells called acute myeloid leukemia.

Administered through subcutaneous or intravenous injection, azacitidine is used for treatment of all five MDS subtypes (according to FAB classification): refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) if accompanied by neutropenia or thrombocytopenia or requiring transfusions, refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL).

Azacitidine belongs to the class of substances termed antimetabolites. Antimetabolites are drugs that interfere with the function of enzymes involved in DNA synthesis.

 

Azacitidine was approved by the FDA in 2004 under the trade name Vidaza as “the first effective treatment for patients with Myelodysplastic Syndrome (MDS).” It is designated as an orphan drug. Azacitidine is synthesized through a multi-step chemical process.