Vidaza (azacitidine)
An approved treatment for Acute Myeloid Leukemia.
The same compound appears under different names depending on the context. Here is how to identify Azacitidine wherever you encounter it, plus the key facts at a glance.
- Generic name
- Azacitidine
- Brand name
- Vidaza
- Development code
- 5-azacytidine
- Drug class
- Hypomethylating agent (nucleoside metabolic inhibitor)
- Manufacturer
- Bristol Myers Squibb (Vidaza); generic manufacturers
- How it's taken
- Subcutaneous injection or IV infusion, 7 days of each 28-day cycle
An injection given for 7 days of each 28-day cycle that is the most common partner for Venclexta in older or unfit adults with AML. Its own FDA label covers myelodysplastic syndromes and juvenile myelomonocytic leukemia, not AML; the AML use rests on the Venclexta label and the VIALE-A trial, so it is listed here as off label.
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Where Azacitidine fits
The usual partner of venetoclax in first-line treatment of older or unfit adults, and of Tibsovo in IDH1-mutated disease; also used alone when venetoclax cannot be given.
How Azacitidine works
Azacitidine is built into DNA and RNA as cells divide. There it traps and destroys DNA methyltransferase, the enzyme that adds methyl tags that silence genes, so genes that control maturation and cell death can be expressed again; at higher concentrations it is directly toxic to fast-dividing cells[1].
Mechanism: Hypomethylating agent: a cytidine analog that blocks DNA methyltransferase, switching silenced genes back on and killing dividing leukemia cells
Side effects and safety
Vidaza has no boxed warning. Its warnings are low blood counts (anemia, neutropenia and thrombocytopenia are expected and counts are checked before each cycle), liver toxicity in people with existing liver disease, kidney toxicity, tumor lysis syndrome and harm to an unborn baby[1]. The most common side effects in its MDS trials were nausea, anemia, thrombocytopenia, vomiting, fever, leukopenia, diarrhea, injection site redness, constipation, neutropenia and bruising[1].
When it is given with Venclexta, the side effect profile is that of the combination, with febrile neutropenia in 42% of VIALE-A patients[2].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Azacitidine
In the VIALE-A regimen azacitidine is given at 75 mg/m2 by injection under the skin or by IV infusion once a day on days 1 to 7 of each 28-day cycle, alongside daily Venclexta[2]. The same 7-day schedule is the label dose for MDS, where at least 4 to 6 cycles are recommended before judging response[1]. It is given at an infusion center or clinic.
Availability and cost
Generic versions may be available at a lower cost. Ask your pharmacist.
Access and eligibility
Coverage help and co-pay or financial support options for Bristol Myers Squibb medicines; generic azacitidine is priced separately and is not covered by the brand program.
Visit BMS Access SupportThe Vidaza label indication is MDS (FAB subtypes) and newly diagnosed JMML in children. In AML it is used as the partner drug named in the Venclexta label and in the Tibsovo label, which is off label for azacitidine itself.
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
Development history
Vidaza was the first hypomethylating agent approved by the FDA, on May 19, 2004, for MDS; a JMML indication was added in May 2022. Generic azacitidine for injection has been available for more than a decade[3].
Explore Acute Myeloid Leukemia trials
Other Acute Myeloid Leukemia treatments
Common questions about Azacitidine
▸What is Azacitidine (Vidaza)?
An injection given for 7 days of each 28-day cycle that is the most common partner for Venclexta in older or unfit adults with AML. Its own FDA label covers myelodysplastic syndromes and juvenile myelomonocytic leukemia, not AML; the AML use rests on the Venclexta label and the VIALE-A trial, so it is listed here as off label.
▸How does Azacitidine work?
Azacitidine is built into DNA and RNA as cells divide. There it traps and destroys DNA methyltransferase, the enzyme that adds methyl tags that silence genes, so genes that control maturation and cell death can be expressed again; at higher concentrations it is directly toxic to fast-dividing cells[1].
▸What are the side effects of Azacitidine?
Vidaza has no boxed warning. Its warnings are low blood counts (anemia, neutropenia and thrombocytopenia are expected and counts are checked before each cycle), liver toxicity in people with existing liver disease, kidney toxicity, tumor lysis syndrome and harm to an unborn baby[1]. The most common side effects in its MDS trials were nausea, anemia, thrombocytopenia, vomiting, fever, leukopenia, diarrhea, injection site redness, constipation, neutropenia and bruising[1]. When it is given with Venclexta, the side effect profile is that of the combination, with febrile neutropenia in 42% of VIALE-A patients[2].
▸How is Azacitidine taken?
In the VIALE-A regimen azacitidine is given at 75 mg/m2 by injection under the skin or by IV infusion once a day on days 1 to 7 of each 28-day cycle, alongside daily Venclexta[2]. The same 7-day schedule is the label dose for MDS, where at least 4 to 6 cycles are recommended before judging response[1]. It is given at an infusion center or clinic.
▸Is Azacitidine FDA approved?
Yes, Azacitidine (Vidaza) is FDA approved (2004) for the treatment of Acute Myeloid Leukemia.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Celgene Corporation via openFDA · 2024-01-29. Vidaza (azacitidine for injection) prescribing information. https://api.fda.gov/drug/label.json?search=set_id:%223495a71a-cc04-4776-851f-f185956f32af%22
- AbbVie Inc. via openFDA · 2026-05-20. Venclexta (venetoclax tablets) prescribing information. https://api.fda.gov/drug/label.json?search=set_id:%22b118a40d-6b56-cee3-10f6-ded821a97018%22
- FDA. Vidaza approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=050794