Overview
Azacitidine is a pyrimidine nucleoside analogue with anti-neoplastic activity. It differs from cytosine by the presence of nitrogen in the C5-position, key in its hypomethylating activity. Two main mechanisms of action have been proposed for azacitidine. One of them is the induction of cytotoxicity. As an analogue of cytidine, it is able to incorporate into RNA and DNA, disrupting RNA metabolism and inhibiting protein and DNA synthesis. The other one is through the inhibition of DNA methyltransferase, impairing DNA methylation. Due to its anti-neoplastic activity and its ability to inhibit methylation in replicating DNA, azacytidine has been used mainly used in the treatment of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), two types of cancer characterized by the presence of aberrant DNA methylation. In May 2004, the FDA approved the use of azacitidine administered subcutaneously for the treatment of MDS of all French-American-British (FAB) subtypes. In January 2007, the FDA approved the intravenous administration of azacitidine. The use of oral azacitidine for the treatment of AML in patients in complete remission was approved by the FDA in September 2020.
Indication
Azacitidine (for subcutaneous or intravenous use) is indicated for the treatment of adult patients with the following French-American-British (FAB) myelodysplastic syndrome (MDS) subtypes: refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL). Azacitidine is also indicated for the treatment of pediatric patients aged 1 month and older with newly diagnosed Juvenile Myelomonocytic Leukemia (JMML). Azacitidine (for oral use) is indicated for continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission or complete remission with incomplete blood count recovery following intensive induction chemotherapy and are not able to complete intensive curative therapy.
Associated Conditions
- Acute Myeloid Leukemia
- Chronic Myelomonocytic Leukemia
- Refractory Anemia
- Refractory Anemia With Excess Blasts in Transformation
- Refractory Anemia With Excess of Blasts (RAEB)
- Refractory Anemia With Ringed Sideroblasts
- Newly diagnosed Juvenile Myelomonocytic Leukaemias (JMML)
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/07/11 | Not Applicable | Not yet recruiting | |||
2025/07/01 | Not Applicable | Not yet recruiting | |||
2025/06/12 | Phase 1 | Not yet recruiting | The Lymphoma Academic Research Organisation | ||
2025/06/10 | Phase 2 | Recruiting | yuejun Liu | ||
2025/06/05 | Phase 3 | Not yet recruiting | |||
2025/04/15 | Phase 3 | Not yet recruiting | |||
2025/04/01 | Phase 2 | Recruiting | |||
2025/03/26 | Phase 1 | ENROLLING_BY_INVITATION | Andrew P. Groves | ||
2025/02/28 | Phase 3 | Recruiting | |||
2025/01/20 | Phase 2 | Recruiting | Justin Watts, MD |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Sandoz Inc | 0781-3253 | SUBCUTANEOUS, INTRAVENOUS | 100 mg in 1 1 | 6/9/2022 | |
| Celgene Corporation | 59572-730 | ORAL | 200 mg in 1 1 | 10/26/2022 | |
| Dr. Reddy's Laboratories Inc. | 43598-143 | SUBCUTANEOUS, INTRAVENOUS | 100 mg in 50 mL | 8/30/2018 | |
| NorthStar RxLLC | 16714-927 | SUBCUTANEOUS, INTRAVENOUS | 100 mg in 1 1 | 12/26/2023 | |
| CELLTRION USA, INC. | 72606-558 | INTRAVENOUS, SUBCUTANEOUS | 100 mg in 1 1 | 1/7/2020 | |
| Sandoz Inc | 0781-9253 | SUBCUTANEOUS, INTRAVENOUS | 100 mg in 1 1 | 9/23/2022 | |
| Fresenius Kabi USA, LLC | 63323-771 | INTRAVENOUS, SUBCUTANEOUS | 100 mg in 30 mL | 9/30/2018 | |
| Camber Pharmaceuticals, Inc. | 31722-365 | INTRAVENOUS, SUBCUTANEOUS | 100 mg in 1 1 | 4/8/2025 | |
| Armas Pharmaceuticals Inc. | 72485-201 | INTRAVENOUS, SUBCUTANEOUS | 100 mg in 1 1 | 12/22/2020 | |
| Accord Healthcare Inc. | 16729-306 | INTRAVENOUS, SUBCUTANEOUS | 100 mg in 1 1 | 1/4/2024 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 2/13/2020 | ||
Authorised | 3/24/2020 | ||
Authorised | 12/17/2008 | ||
Authorised | 3/27/2020 | ||
Authorised | 1/5/2024 | ||
Authorised | 6/17/2021 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| ONUREG FILM-COATED TABLETS 300 MG | SIN16573P | TABLET, FILM COATED | 300.0 mg | 8/19/2022 | |
| AZACITIDINE ADVAGEN POWDER FOR SUSPENSION FOR INJECTION 100MG/VIAL | SIN16285P | INJECTION, POWDER, FOR SUSPENSION | 100mg/vial | 7/23/2021 | |
| Vidaza powder for suspension for injection 100mg/vial | SIN13801P | INJECTION, POWDER, FOR SUSPENSION | 100mg/vial | 5/18/2010 | |
| XPREZA 100 INJECTION 100 MG/VIAL | SIN15118P | INJECTION | 100mg | 11/23/2016 | |
| AVOXRED POWDER FOR SUSPENSION FOR INJECTION 100MG/VIAL | SIN15875P | INJECTION, POWDER, LYOPHILIZED, FOR SUSPENSION | 100 mg/ml | 1/16/2020 | |
| ONUREG FILM-COATED TABLETS 200 MG | SIN16572P | TABLET, FILM COATED | 200.0 mg | 8/19/2022 | |
| AZADINE POWDER FOR SUSPENSION FOR INJECTION 100MG/VIAL | SIN15304P | INJECTION, POWDER, LYOPHILIZED, FOR SUSPENSION | 100 mg | 7/25/2017 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| Azacitidine for Injection | 国药准字H20213659 | 化学药品 | 注射剂 | 8/25/2021 | |
| Azacitidine for Injection | 国药准字H20213493 | 化学药品 | 注射剂 | 6/16/2021 | |
| Azacitidine for Injection | 国药准字H20193278 | 化学药品 | 注射剂 | 9/26/2019 | |
| Azacitidine for Injection | 国药准字H20243174 | 化学药品 | 注射剂 | 2/6/2024 | |
| Azacitidine for Injection | 国药准字HJ20170238 | 化学药品 | 注射剂 | 2/14/2022 | |
| Azacitidine for Injection | 国药准字H20234059 | 化学药品 | 注射剂 | 8/29/2023 | |
| Azacitidine for Injection | 国药准字H20233219 | 化学药品 | 注射剂 | 2/28/2023 | |
| Azacitidine for Injection | 国药准字H20244451 | 化学药品 | 注射剂 | 7/16/2024 | |
| Azacitidine for Injection | 国药准字H20193280 | 化学药品 | 注射剂 | 4/7/2024 |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| AZACITIDINE SANDOZ POWDER FOR SUSPENSION FOR INJECTION 100MG | N/A | N/A | N/A | 12/13/2024 |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| AZACITIDINE-TEVA azacitidine 100 mg powder for injection vial | 299040 | Medicine | A | 7/23/2019 | |
| AZACITIDINE EPSL azacitidine 100 mg powder for injection vial | 407492 | Medicine | A | 2/23/2024 | |
| REDDAZA azacitidine 100 mg powder for injection vial | 231946 | Medicine | A | 2/10/2016 | |
| AZACITIDINE ANS azacitidine 100 mg powder for injection vial | 395836 | Medicine | A | 12/22/2023 | |
| ONUREG azacitidine 300 mg film-coated tablet blister pack | 370372 | Medicine | A | 4/8/2022 | |
| AZACCORD azacitidine 100mg powder for injection vial | 265773 | Medicine | A | 5/8/2017 | |
| AZACITIDINE INTAS azacitidine 100mg powder for injection vial | 265775 | Medicine | A | 5/8/2017 | |
| Azacitidine APOTEX azacitidine100 mg powder for injection vial | 231497 | Medicine | A | 9/23/2015 | |
| AZACITIDINE SANDOZ azacitidine 100 mg powder for injection vial | 338376 | Medicine | A | 10/21/2021 | |
| ONUREG azacitidine 200 mg film-coated tablet blister pack | 370367 | Medicine | A | 4/8/2022 |
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