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)

CountryAccess RouteRegulatory PathwayLead TimeStatus

Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Azacitidine
Generic NameAzacitidine
Brands1 brand available
Active IngredientAzacitidine
Drug ClassMyelodysplastic syndromes
ManufacturerCelgene
Dosage FormsSubcutaneous injection, 100mg vial; IV infusion
Medical CodeL01BC07
Orphan StatusYes — Orphan Drug
Cold ChainRequired
Lead Time5 days
Reg. StatusFDA Approved
Clinical TrialNCT00071799
Countries8 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes