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Cytosine Arabinoside

University of Rochester · FDA-approved active Small molecule

Cytosine Arabinoside is a Nucleoside analog antimetabolite Small molecule drug developed by University of Rochester. It is currently FDA-approved for Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL), Chronic myeloid leukemia (CML) in blast crisis. Also known as: AraC, Cytosar U, Cytosine aribinoside, Arabinosylcytosine.

Cytosine arabinoside is a nucleoside analog that inhibits DNA synthesis by being incorporated into DNA and blocking DNA polymerase, leading to cell death.

Cytosine arabinoside is a nucleoside analog that inhibits DNA synthesis by being incorporated into DNA and blocking DNA polymerase, leading to cell death. Used for Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL), Chronic myeloid leukemia (CML) in blast crisis.

At a glance

Generic nameCytosine Arabinoside
Also known asAraC, Cytosar U, Cytosine aribinoside, Arabinosylcytosine, Cytarabine sterile
SponsorUniversity of Rochester
Drug classNucleoside analog antimetabolite
TargetDNA polymerase; incorporated into DNA
ModalitySmall molecule
Therapeutic areaOncology
PhaseFDA-approved

Mechanism of action

Cytosine arabinoside (ara-C) is a cytidine analog that enters cells and is phosphorylated to its active triphosphate form. It then incorporates into DNA during replication, causing chain termination and inhibiting DNA synthesis. This mechanism is particularly effective against rapidly dividing cells, making it useful in hematologic malignancies where leukemic blasts are highly proliferative.

Approved indications

Common side effects

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

Frequently asked questions about Cytosine Arabinoside

What is Cytosine Arabinoside?

Cytosine Arabinoside is a Nucleoside analog antimetabolite drug developed by University of Rochester, indicated for Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL), Chronic myeloid leukemia (CML) in blast crisis.

How does Cytosine Arabinoside work?

Cytosine arabinoside is a nucleoside analog that inhibits DNA synthesis by being incorporated into DNA and blocking DNA polymerase, leading to cell death.

What is Cytosine Arabinoside used for?

Cytosine Arabinoside is indicated for Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL), Chronic myeloid leukemia (CML) in blast crisis, Lymphoma.

Who makes Cytosine Arabinoside?

Cytosine Arabinoside is developed and marketed by University of Rochester (see full University of Rochester pipeline at /company/university-of-rochester).

Is Cytosine Arabinoside also known as anything else?

Cytosine Arabinoside is also known as AraC, Cytosar U, Cytosine aribinoside, Arabinosylcytosine, Cytarabine sterile.

What drug class is Cytosine Arabinoside in?

Cytosine Arabinoside belongs to the Nucleoside analog antimetabolite class. See all Nucleoside analog antimetabolite drugs at /class/nucleoside-analog-antimetabolite.

What development phase is Cytosine Arabinoside in?

Cytosine Arabinoside is FDA-approved (marketed).

What are the side effects of Cytosine Arabinoside?

Common side effects of Cytosine Arabinoside include Myelosuppression (neutropenia, thrombocytopenia, anemia), Nausea and vomiting, Mucositis, Hepatotoxicity, Neurotoxicity (at high doses), Infection.

What does Cytosine Arabinoside target?

Cytosine Arabinoside targets DNA polymerase; incorporated into DNA and is a Nucleoside analog antimetabolite.

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