U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ACHIRAL
Molecular Formula CH4N2O2
Molecular Weight 76.0547
Optical Activity NONE
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of Hydroxyurea

SMILES

NC(=O)NO

InChI

InChIKey=VSNHCAURESNICA-UHFFFAOYSA-N
InChI=1S/CH4N2O2/c2-1(4)3-5/h5H,(H3,2,3,4)

HIDE SMILES / InChI
Hydroxyurea is an oral antimetabolite; inhibits ribonucleotide reductase and DNA synthesis. It is used for resistant chronic myeloid leukemia, locally advanced squamous cell carcinomas of the head and neck (excluding lip) in combination with concurrent chemoradiation, and to reduce the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises. Hydroxyurea, a myelosuppressive agent, is the only effective drug proven to reduce the frequency of painful episodes. It raises the level of HbF and the haemoglobin level. It usually decreases the rate of painful episodes by 50%. It was first tested in sickle cell disease in 1984. It also decreases the rate of ACS episodes and blood transfusions by ~50 % in adults. It was developed as an anticancer drug and has been used to treat myeloproliferative syndromes-leukemia, melanoma, and ovarian cancer. It was approved for use by FDA in adults. Side effects includes neutropenia, bone marrow suppression, elevation of hepatic enzymes, anorexia, nausea, vomiting and infertility.

Approval Year

Targets

Targets

Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
DROXIA

Approved Use

DROXIA is an antimetabolite indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises

Launch Date

1967
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
793.75 μM
2000 mg single, oral
dose: 2000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
HYDROXYUREA plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
3934 μM × h
2000 mg single, oral
dose: 2000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
HYDROXYUREA plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
3.32 h
2000 mg single, oral
dose: 2000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
HYDROXYUREA plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
Doses

Doses

DosePopulationAdverse events​
40 mg/kg 2 times / day single, oral
Highest studied dose
Dose: 40 mg/kg, 2 times / day
Route: oral
Route: single
Dose: 40 mg/kg, 2 times / day
Sources:
unhealthy, 2-18 years
n = 10
Health Status: unhealthy
Condition: allogeneic transplantation
Age Group: 2-18 years
Population Size: 10
Sources:
26.7 mg/kg 1 times / day multiple, oral
MTD
Dose: 26.7 mg/kg, 1 times / day
Route: oral
Route: multiple
Dose: 26.7 mg/kg, 1 times / day
Sources:
unhealthy, 2-18 years
Health Status: unhealthy
Condition: sickle cell anemia
Age Group: 2-18 years
Sources:
20 mg/kg 1 times / day multiple, oral
Recommended
Dose: 20 mg/kg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg/kg, 1 times / day
Sources:
unhealthy, 2-18 years
Health Status: unhealthy
Age Group: 2-18 years
Sources:
Other AEs: Myelosuppression...
20 mg/kg 1 times / day steady, oral
Dose: 20 mg/kg, 1 times / day
Route: oral
Route: steady
Dose: 20 mg/kg, 1 times / day
Sources:
unhealthy
n = 104
Health Status: unhealthy
Condition: Malaria
Population Size: 104
Sources:
Other AEs: Sudden death, Thrombocytopenia...
Other AEs:
Sudden death (serious, 1 patient)
Thrombocytopenia (below serious, 11 patient)
Sources:
AEs

AEs

AESignificanceDosePopulation
Myelosuppression
20 mg/kg 1 times / day multiple, oral
Recommended
Dose: 20 mg/kg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg/kg, 1 times / day
Sources:
unhealthy, 2-18 years
Health Status: unhealthy
Age Group: 2-18 years
Sources:
Thrombocytopenia below serious, 11 patient
20 mg/kg 1 times / day steady, oral
Dose: 20 mg/kg, 1 times / day
Route: oral
Route: steady
Dose: 20 mg/kg, 1 times / day
Sources:
unhealthy
n = 104
Health Status: unhealthy
Condition: Malaria
Population Size: 104
Sources:
Sudden death serious, 1 patient
20 mg/kg 1 times / day steady, oral
Dose: 20 mg/kg, 1 times / day
Route: oral
Route: steady
Dose: 20 mg/kg, 1 times / day
Sources:
unhealthy
n = 104
Health Status: unhealthy
Condition: Malaria
Population Size: 104
Sources:
Overview

Overview

Drug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
likely [IC50 >10 uM]
no [Activation 39.8107 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >133 uM]
no [IC50 >133 uM]
no [IC50 >133 uM]
no [IC50 >133 uM]
no
no
yes
yes
yes
Drug as victimTox targets
PubMed

PubMed

TitleDatePubMed
Senescence-like changes induced by hydroxyurea in human diploid fibroblasts.
2000 Aug
Hydroxyurea induces a senescence-like change of K562 human erythroleukemia cell.
2000 Aug
A case of bilateral heel ulcers associated with hydroxyurea therapy for chronic myelogenous leukemia.
2000 Mar
Dermatomyositis-like eruption following hydroxyurea therapy.
2000 May
Long-term hydroxyurea in combination with didanosine and stavudine for the treatment of HIV-1 infection. Swiss HIV Cohort Study.
2000 Sep 29
[Low-dose cytosine-arabinoside (Ara-C) therapy for chronic myeloid leukemia].
2001
Systematic review of combination antiretroviral therapy with didanosine plus hydroxyurea: a partial solution to Africa's HIV/AIDS problem?
2001
Hydroxyurea-induced oxidative damage of normal and sickle cell hemoglobins in vitro: amelioration by radical scavengers.
2001
Sorting of plant chromosomes.
2001
Cutaneous adverse reactions to hydroxyurea in patients with sickle cell disease.
2001 Apr
A new molecular role for iron in regulation of cell cycling and differentiation of HL-60 human leukemia cells: iron is required for transcription of p21(WAF1/CIP1) in cells induced by phorbol myristate acetate.
2001 Apr
The Saccharomyces cerevisiae phosphotyrosyl phosphatase activator proteins are required for a subset of the functions disrupted by protein phosphatase 2A mutations.
2001 Apr 1
Role of petasin in the potential anti-inflammatory activity of a plant extract of petasites hybridus.
2001 Apr 15
Cell cycle expression of histone genes in Trypanosoma cruzi.
2001 Apr 6
Hydroxyurea promotes the reduction of spontaneous BFU-e to normal levels in SS and S/beta thalassemic patients.
2001 Feb
The predictive value of vascular risk factors and gender for the development of thrombotic complications in essential thrombocythemia.
2001 Feb
Induction of unscheduled DNA synthesis in hairless mouse epidermis by 8-methoxypsoralen plus ultraviolet A (PUVA).
2001 Feb
Foraging behaviour in Drosophila larvae: mushroom body ablation.
2001 Feb
Improved cerebrovascular patency following therapy in patients with sickle cell disease: initial results in 4 patients who received HLA-identical hematopoietic stem cell allografts.
2001 Feb
The potential place of chloroquine in the treatment of HIV-1-infected patients.
2001 Feb
Effects of hydroxurea, stem cell factor, and erythropoietin in combination on fetal hemoglobin in the baboon.
2001 Feb
Leukotriene modifiers in pediatric asthma management.
2001 Feb
Tof1p regulates DNA damage responses during S phase in Saccharomyces cerevisiae.
2001 Feb
Coupling of Saccharomyces cerevisiae early meiotic gene expression to DNA replication depends upon RPD3 and SIN3.
2001 Feb
Inhibition of N-myc expression and induction of apoptosis by iron chelation in human neuroblastoma cells.
2001 Feb 1
The biology and treatment of chronic myelogenous leukemia.
2001 Jan
Current management in polycythemia vera.
2001 Jan
Predominantly BCR-ABL negative myeloid precursors in interferon-alpha treated chronic myelogenous leukemia: a follow-up study of peripheral blood colony-forming cells with fluorescence in situ hybridization.
2001 Jan
Effects of amifostine in a patient with an advanced-stage myelodysplastic syndrome.
2001 Jan
Inhibition of hepatitis B virus production associated with high levels of intracellular viral DNA intermediates in iron-depleted HepG2.2.15 cells.
2001 Jan
N-(Hydroxyaminocarbonyl)phenylalanine: a novel class of inhibitor for carboxypeptidase A.
2001 Jan
Cell division activity during apical hook development.
2001 Jan
Thioguanine for refractory psoriasis: a 4-year experience.
2001 Jan
Requirement for three novel protein complexes in the absence of the Sgs1 DNA helicase in Saccharomyces cerevisiae.
2001 Jan
Drug Points: pancreatitis associated with hydroxyurea in combination with didanosine.
2001 Jan 13
De novo activation of the beta-phaseolin promoter by phosphatase or protein synthesis inhibitors.
2001 Jan 19
JP-8 jet fuel-induced DNA damage in H4IIE rat hepatoma cells.
2001 Jan 25
Reduction of telomerase activity in human liver cancer cells by a histone deacetylase inhibitor.
2001 Jun
[Hydroxyurea--is it a harmless drug in Vaquez disease?]].
2001 Mar
Muco-cutaneous changes during long-term therapy with hydroxyurea in chronic myeloid leukaemia.
2001 Mar
Localised chronic eyedlid disease resulting from long term hydroxyurea therapy.
2001 Mar
Prognostic impact of bone marrow erythropoietic precursor cells and myelofibrosis at diagnosis of Ph1+ chronic myelogenous leukaemia--a multicentre study on 495 patients.
2001 Mar
Synergistic activity of the new ABL-specific tyrosine kinase inhibitor STI571 and chemotherapeutic drugs on BCR-ABL-positive chronic myelogenous leukemia cells.
2001 Mar
A unique, complex variant philadelphia chromosome translocation in a patient with typical chronic myelogenous leukemia.
2001 Mar
Pharmacologic induction of fetal hemoglobin: raising the therapeutic bar in sickle cell disease.
2001 Mar
Topoisomerase IV, alone, unknots DNA in E. coli.
2001 Mar 15
Osteoclast differentiation factor modulates cell cycle machinery and causes a delay in s phase progression in RAW264 cells.
2001 Mar 23
N-Aminoindoline derivatives as inhibitors of 5-lipoxygenase.
2001 Mar 26
The BARD1-CstF-50 interaction links mRNA 3' end formation to DNA damage and tumor suppression.
2001 Mar 9
Skin and nail changes in children with sickle cell anemia receiving hydroxyurea therapy.
2001 May
Patents

Sample Use Guides

Usual Adult Dose for Chronic Myelogenous Leukemia 15 mg/kg/day orally as a single dose Usual Adult Dose for Head and Neck Cancer 15 mg/kg/day orally as a single dose Usual Adult Dose for Sickle Cell Anemia 15 mg/kg orally once a day; increase 5 mg/kg/day every 12 weeks Maximum dose: 35 mg/kg/day Usual Pediatric Dose for Sickle Cell Anemia 2 years and older: 20 mg/kg orally once a day; increase 5 mg/kg/day every 8 weeks or if a painful crisis occurs; increase dosing only if blood counts are in the acceptable range; do not increase dosing if myelosuppression occurs; if blood counts are considered toxic, discontinue therapy until hematologic recovery Duration of therapy: Give until mild myelosuppression (absolute neutrophil count 2000/microliter to 4000/microliter) is achieved, up to 35 mg/kg/day Maximum dose: 35 mg/kg/day
Route of Administration: Oral
Name Type Language
Hydroxyurea
HSDB   MI   ORANGE BOOK   USAN   USP   USP-RS   VANDF  
USAN  
Official Name English
HYDROXYUREA [HSDB]
Common Name English
SIKLOS
Brand Name English
Hydroxycarbamide [WHO-DD]
Common Name English
SQ 1089
Code English
HYDREA
Brand Name English
HYDROXYCARBAMIDE [EMA EPAR]
Common Name English
HYDROXYCARBAMIDE
EMA EPAR   EP   INN   JAN   MART.   WHO-DD  
INN  
Official Name English
HYDROXYUREA [VANDF]
Common Name English
SQ-1089
Code English
UREA, HYDROXY-
Systematic Name English
hydroxycarbamide [INN]
Common Name English
HYDROXYUREA [IARC]
Common Name English
HYDROXYUREA [USP-RS]
Common Name English
HYDROXYUREA [USP MONOGRAPH]
Common Name English
DROXIA
Brand Name English
HYDROXYCARBAMIDE [EP MONOGRAPH]
Common Name English
HYDROXYCARBAMIDE [JAN]
Common Name English
NSC-32065
Code English
HYDROXYUREA [MI]
Common Name English
HYDROXYUREA [ORANGE BOOK]
Common Name English
HYDROXYUREA [USAN]
Common Name English
HYDROXYCARBAMIDE [MART.]
Common Name English
Classification Tree Code System Code
WHO-ESSENTIAL MEDICINES LIST 8.2
Created by admin on Fri Dec 15 15:07:48 GMT 2023 , Edited by admin on Fri Dec 15 15:07:48 GMT 2023
WHO-ATC L01XX05
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FDA ORPHAN DRUG 202605
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WHO-ESSENTIAL MEDICINES LIST 10.3
Created by admin on Fri Dec 15 15:07:48 GMT 2023 , Edited by admin on Fri Dec 15 15:07:48 GMT 2023
FDA ORPHAN DRUG 555116
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NCI_THESAURUS C2150
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NDF-RT N0000006999
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LIVERTOX NBK548724
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FDA ORPHAN DRUG 463414
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NDF-RT N0000180853
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EU-Orphan Drug EU/3/03/154
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NCI_THESAURUS C272
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EMA ASSESSMENT REPORTS SIKLOS (AUTHORIZED: ANEMIA, SICKLE-CELL)
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FDA ORPHAN DRUG 400413
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FDA ORPHAN DRUG 49590
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WHO-VATC QL01XX05
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Code System Code Type Description
CHEBI
44423
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PRIMARY
RXCUI
5552
Created by admin on Fri Dec 15 15:07:48 GMT 2023 , Edited by admin on Fri Dec 15 15:07:48 GMT 2023
PRIMARY RxNorm
ECHA (EC/EINECS)
204-821-7
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PRIMARY
LACTMED
Hydroxyurea
Created by admin on Fri Dec 15 15:07:48 GMT 2023 , Edited by admin on Fri Dec 15 15:07:48 GMT 2023
PRIMARY
HSDB
6887
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PRIMARY
DRUG BANK
DB01005
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PRIMARY
CAS
127-07-1
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PRIMARY
NSC
32065
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PRIMARY
SMS_ID
100000085457
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PRIMARY
INN
1991
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PRIMARY
NCI_THESAURUS
C560
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PRIMARY
MESH
D006918
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PRIMARY
WIKIPEDIA
HYDROXYCARBAMIDE
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PRIMARY
EVMPD
SUB08076MIG
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PRIMARY
ChEMBL
CHEMBL467
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PRIMARY
DAILYMED
X6Q56QN5QC
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PRIMARY
PUBCHEM
3657
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PRIMARY
MERCK INDEX
m6158
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PRIMARY Merck Index
EPA CompTox
DTXSID6025438
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PRIMARY
DRUG CENTRAL
1399
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PRIMARY
IUPHAR
6822
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PRIMARY
FDA UNII
X6Q56QN5QC
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PRIMARY
RS_ITEM_NUM
1332000
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PRIMARY