Azacitidine
Azacitidine is an antineoplastic medication used primarily to treat myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and certain types of acute myeloid leukemia (AML). As a pyrimidine nucleoside analog, it works by causing hypomethylation of DNA and directly targeting abnormal hematopoietic cells in the bone marrow, helping to restore normal cell differentiation and proliferation.
Azacitidine is a chemotherapy agent that treats blood disorders like MDS by reprogramming abnormal bone marrow cells through DNA hypomethylation.
Key facts at a glance
- ATC
- L01BC07
- RxNorm
- 1251
- Generic
- Azacitidine
- Class
- atc-l01
- Brands
- Onureg, Vidaza
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- injection, tablet
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
9 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks to the fetus
regular fetal monitoring and ultrasound examinations
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks to the fetus
regular fetal monitoring and ultrasound examinations
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring and assessment of fetal well-being
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingdiscontinue breastfeeding
do not breastfeed during treatment and for at least 1 week after last dose
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
pediatric_age
children under 18not established
safety and efficacy not established in pediatric patients
not applicable
geriatric
elderly 65 and olderno specific dose adjustment required
use with caution due to potential decreased renal function; monitor renal function
monitor renal function and blood counts regularly
Use with caution and monitor for adverse effects.
renal_impairment
mild to moderateno initial dose adjustment required
use with caution; monitor renal function
monitor renal function and hematologic parameters
Use with caution and monitor for adverse effects.
renal_impairment
severereduce dose
reduce starting dose; use with extreme caution
frequent monitoring of renal function and blood counts
Use of this medication in this population requires careful consideration of risks and benefits.
hepatic_impairment
mild to moderateno specific dose adjustment required
use with caution; monitor liver function tests
monitor liver function tests regularly
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Azacitidine is classified as a hypomethylating agent and antineoplastic medication
Common uses
Primary therapeutic indications for Azacitidine
FDA approval year
FDA approval dates for Azacitidine formulations
Mechanism of action
Primary pharmacological mechanism
Azacitidine is indicated for the treatment of myelodysplastic syndromes (MDS) of various subtypes, including refractory anemia with excess blasts (RAEB), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML) with 20-30% blasts in patients who are not eligible for hematopoietic stem cell transplantation.
Azacitidine is a pyrimidine nucleoside analog of cytidine that exerts its effects through two mechanisms: (1) hypomethylation of DNA by inhibiting DNA methyltransferases, which can reactivate tumor suppressor genes and restore normal cell differentiation; and (2) direct cytotoxicity on rapidly dividing abnormal hematopoietic cells in the bone marrow.
Azacitidine is administered by subcutaneous injection or intravenous infusion, typically in 7-day cycles repeated every 4 weeks. Common side effects include bone marrow suppression (anemia, neutropenia, thrombocytopenia), nausea, vomiting, and injection site reactions. Treatment should be supervised by oncologists or hematologists experienced in chemotherapy, with regular monitoring of blood counts. It is contraindicated in patients with hypersensitivity to the drug and should be used with caution in patients with severe hepatic or renal impairment.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy