Azacitidine
Orphan Drug Cold Chain RequiredFDA Approved
Description
Azacitidine is a hypomethylating agent used in the treatment of myelodysplastic syndromes and acute myeloid leukemia. It works by inhibiting DNA methyltransferase, leading to hypomethylation of DNA and direct cytotoxicity on abnormal hematopoietic cells. The drug has shown efficacy in treatment-related myelodysplastic syndromes including those related to prior chemotherapy exposure.
Indications & Therapeutic Use
Myelodysplastic syndromes, acute myeloid leukemia with 20-30% blasts, chronic myelomonocytic leukemia
Global Availability (8 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Azacitidine
| Generic Name | Azacitidine |
| Brands | 1 brand available |
| Active Ingredient | Azacitidine |
| Drug Class | Myelodysplastic syndromes |
| Manufacturer | Celgene |
| Dosage Forms | Subcutaneous injection, 100mg vial; IV infusion |
| Medical Code | L01BC07 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 5 days |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT00071799 |
| Countries | 8 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes