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Azacitidine

Generic Name
Azacitidine
Brand Names
Onureg, Vidaza, Azacitidine Accord, Azacitidine betapharm, Azacitidine Mylan, Azacitidine Kabi
Drug Type
Small Molecule
Chemical Formula
C8H12N4O5
CAS Number
320-67-2
Unique Ingredient Identifier
M801H13NRU

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.

Background

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
2025/07/01
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
2025/01/16
Phase 2
Not yet recruiting
Daping Hospital and the Research Institute of Surgery of the Third Military Medical University

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

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
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