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
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)
Associated Therapies
-

Safety and Efficacy Study of Pracinostat With Azacitadine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemia (AML)

Phase 2
Completed
Conditions
Interventions
First Posted Date
2013-07-31
Last Posted Date
2021-02-09
Lead Sponsor
Helsinn Healthcare SA
Target Recruit Count
50
Registration Number
NCT01912274
Locations
🇺🇸

The University of Chicago, Chicago, Illinois, United States

🇺🇸

MD Anderson, Houston, Texas, United States

🇺🇸

University of Kansas Cancer Center, Westwood, Kansas, United States

and more 14 locations

Azacitidine and Entinostat in Treating Patients With Newly Diagnosed Stage IA-IIIA Non-Small Lung Cancer Undergoing Surgery

First Posted Date
2013-06-26
Last Posted Date
2016-11-18
Lead Sponsor
National Cancer Institute (NCI)
Target Recruit Count
40
Registration Number
NCT01886573
Locations
🇺🇸

University of New Mexico, Albuquerque, New Mexico, United States

🇺🇸

University of Texas Southwestern Medical Center, Dallas, Texas, United States

🇺🇸

University of Kentucky/Markey Cancer Center, Lexington, Kentucky, United States

Phase 2 Study of Pracinostat With Azacitidine in Patients With Previously Untreated Myelodysplastic Syndrome

First Posted Date
2013-06-10
Last Posted Date
2018-09-13
Lead Sponsor
Helsinn Healthcare SA
Target Recruit Count
102
Registration Number
NCT01873703
Locations
🇺🇸

Woodlands Medical Specialists, Pensacola, Florida, United States

🇺🇸

Weill Cornell Medical Center, New York, New York, United States

🇺🇸

Florida Cancer Specialists North, Saint Petersburg, Florida, United States

and more 21 locations

A Phase I Study of 5-Azacytidine in Combination With Chemotherapy for Children With Relapsed or Refractory ALL or AML

First Posted Date
2013-05-23
Last Posted Date
2021-06-09
Lead Sponsor
Therapeutic Advances in Childhood Leukemia Consortium
Target Recruit Count
15
Registration Number
NCT01861002
Locations
🇺🇸

Children's National Medical Center, Washington, District of Columbia, United States

🇨🇦

Sainte-Justine University Hospital Center, Montreal, Quebec, Canada

🇺🇸

Children's Mercy Hospitals and Clinics, Kansas City, Missouri, United States

and more 20 locations

Study of MLN4924 Plus Azacitidine in Treatment-naive Participants With Acute Myelogenous Leukemia (AML) Who Are 60 Years or Older

Phase 1
Completed
Conditions
Interventions
First Posted Date
2013-03-20
Last Posted Date
2020-03-03
Lead Sponsor
Millennium Pharmaceuticals, Inc.
Target Recruit Count
64
Registration Number
NCT01814826
Locations
🇺🇸

Hospital Corporation of America-HealthOne, LLC, Denver, Colorado, United States

🇺🇸

UNC-Chapel Hill School of Medicine, Chapel Hill, North Carolina, United States

🇺🇸

University of Miami School of Medicine, Miami, Florida, United States

and more 5 locations

Azacitidine Compared to Conventional Chemotherapy as Consolidation of Elderly Patients With AML

Phase 2
Terminated
Conditions
Interventions
First Posted Date
2013-02-18
Last Posted Date
2015-09-22
Lead Sponsor
Karolinska University Hospital
Target Recruit Count
130
Registration Number
NCT01794169
Locations
🇸🇪

Karolinska University Hospital, Solna, Stockholm, Sweden

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