Azacitidine
A gentle chemotherapy that switches silenced genes back on. With venetoclax it became the standard for older people with AML who cannot take intensive treatment.
Approved for MDS (2004) and, in combination with venetoclax, for newly diagnosed AML in patients unfit for intensive chemotherapy (VIALE-A, 2020). Oral azacitidine (Onureg, CC-486) is approved as maintenance after intensive induction (QUAZAR AML-001: OS 24.7 vs 14.8 months). Backbone partner for IDH inhibitors (AGILE), menin inhibitors, and FLT3 inhibitors in trials.
1.Azacitidine enters the cell and is phosphorylated
- Route
- Subcutaneous or IV (Vidaza); oral (Onureg)
- Schedule
- 75 mg/m² daily for 7 days of each 28-day cycle (with venetoclax in AML); Onureg 300 mg daily for 14 of 28 days as maintenance
- Monitoring
- Blood counts each cycle; renal function
Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.
- Medicare
- Part B or Part D
Injectable azacitidine (Vidaza and generics) is a Part B drug given subcutaneously or IV in the clinic. Oral azacitidine (Onureg) has a different indication (AML maintenance) and is a Part D drug.
- Commercial insurance
- covered with prior authorisation
Injectable generic azacitidine is covered on label without much review; Onureg requires prior authorisation and specialty pharmacy dispensing.
- Assistance programmes
- BMS Access Support
- Bristol Myers Squibb Patient Assistance Foundation
- PAN Foundation — Disease-specific co-pay and premium funds; open and closed funds change monthly.
- HealthWell Foundation
- CancerCare Co-Payment Assistance Foundation
- Patient Advocate Foundation Co-Pay Relief
- Leukemia & Lymphoma Society financial support
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
- Appraised for
- Intermediate-2 or high-risk MDS, CMML, and AML with 20-30% blasts not eligible for transplant
- Notes
- AML with more than 30% blasts (TA399, 2016) was not recommended. Oral azacitidine (Onureg) for AML maintenance appraised 2021-22. Generic injectable since 2020.
- NHS England
- Routinely funded for the appraised indication (or via managed access)
Sources: NICE TA218 · SMC advice: azacitidine. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
- 19 May 2004ApprovalUS
First hypomethylating agent approved (MDS)
- 1 Sept 2020ApprovalUS
Onureg maintenance in AML
- 16 Oct 2020ApprovalUS
Venetoclax + azacitidine full approval in unfit AML (VIALE-A)
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2004 | Myelodysplastic syndromes |
| US | 2020 | Newly diagnosed AML unfit for intensive chemotherapy, with venetoclax (VIALE-A) |
| US | 2020 | Oral azacitidine (Onureg) maintenance after intensive induction (QUAZAR AML-001) |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Neutropenia VIALE-A combination arm | — | 42% |
| Febrile neutropenia VIALE-A combination arm | — | 42% |
| Nausea | 44% | — |
| Injection-site reactions subcutaneous route | 30% | — |
Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Landmark trials in OnCo
AGILE showed that for the roughly 6-10% of AML patients with an IDH1 mutation, a targeted doublet produces survival in the range of two years, an outcome previously unimaginable in unfit patients. Ivosidenib-azacitidine is approved and is one option alongside venetoclax-azacitidine for these patients. Which regimen, or triplet, is best for IDH1-mutated disease has not been settled by a randomised trial.
VIALE-A turned a palliative regimen into one that produces remission in two-thirds of older AML patients and is now the reference treatment for anyone not fit for intensive chemotherapy. It shifted the field towards lower-intensity targeted combinations and opened the door to adding FLT3, IDH and menin inhibitors to the backbone. Cure remains uncommon and most patients relapse within two years.
Latest papers
topQuery for this drug: (TITLE:"Azacitidine" OR ABSTRACT:"Azacitidine" OR TITLE:"Vidaza" OR ABSTRACT:"Vidaza" OR TITLE:"Onureg" OR ABSTRACT:"Onureg" OR TITLE:"oral" OR ABSTRACT:"oral") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Azacitidine, not a curated reading list.
Pages like this
not linked directly; found by shared links- ProductDecitabine + cedazuridine (oral)
Shares Shorter venetoclax courses in unfit AML, Venetoclax + hypomethylating agent, Unmask hidden antigens with a short epigenetic course before immunotherapy, Myelodysplastic syndromes / neoplasms (MDS).
- ProductVenetoclax
Shares Shorter venetoclax courses in unfit AML, Hypomethylating agents (azacitidine, decitabine), Venetoclax + hypomethylating agent, 7+3 induction chemotherapy.
- ProductIvosidenib
Shares AGILE, AGILE: ivosidenib plus azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia.
- TargetMenin
Shares Menin inhibitor + venetoclax + azacitidine, Intrinsic apoptosis (BCL-2 family), Epigenetic reprogramming, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET).
- ProductZiftomenib
Shares Menin inhibitor + venetoclax + azacitidine, Epigenetic reprogramming, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia.
- ProductRevumenib
Shares Menin inhibitor + venetoclax + azacitidine, Epigenetic reprogramming, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia.
- ProductEnasidenib
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Bristol Myers Squibb, Acute myeloid leukaemia.
- ProductTreosulfan
Shares Myelodysplastic syndromes / neoplasms (MDS), Cytotoxic chemotherapy, Acute myeloid leukaemia.