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

Details

Stereochemistry ACHIRAL
Molecular Formula C2H7NS
Molecular Weight 77.149
Optical Activity NONE
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of CYSTEAMINE

SMILES

NCCS

InChI

InChIKey=UFULAYFCSOUIOV-UHFFFAOYSA-N
InChI=1S/C2H7NS/c3-1-2-4/h4H,1-3H2

HIDE SMILES / InChI

Molecular Formula C2H7NS
Molecular Weight 77.149
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

Description
Curator's Comment: description was created based on several sources, including https://www.ncbi.nlm.nih.gov/pubmed/6131501 | https://www.ncbi.nlm.nih.gov/pubmed/6124307 | https://www.ncbi.nlm.nih.gov/pubmed/15675041 | https://www.ncbi.nlm.nih.gov/pubmed/28384851

Cysteamine (trade name CYSTAGON) is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis. Cystinosis is an autosomal recessive inborn error of metabolism in which the transport of cystine out of lysosomes is abnormal; in the nephropathic form, accumulation of cystine and formation of crystals damage various organs, especially the kidney, leading to renal tubular Fanconi Syndrome and progressive glomerular failure, with end-stage renal failure by the end of the first decade of life. In four studies of cystinosis patients before cysteamine was available, renal death (need for transplant or dialysis) occurred at the median age of fewer than 10 years. Patients with cystinosis also experience growth failure, rickets, and photophobia due to cystine deposits in the cornea. With time most organs are damaged, including the retina, muscles and central nervous system. Cysteamine is an aminothiol that participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis.

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
Target ID: P21980
Gene ID: 7052.0
Gene Symbol: TGM2
Target Organism: Homo sapiens (Human)
178.0 µM [IC50]
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
CYSTAGON

Approved Use

PROCYSBI is indicated for the treatment of nephropathic cystinosis in adult and pediatric patients 2 years of age and older. PROCYSBI is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adult and pediatric patients 2 years of age and older. (1)

Launch Date

7.7690883E11
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
2.7 mg/L
404 mg/m² 4 times / day steady-state, oral
dose: 404 mg/m²
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
CYSTEAMINE plasma
Homo sapiens
population: UNHEALTHY
age: CHILD
sex: FEMALE / MALE
food status: UNKNOWN
37.72 μM
450 mg single, oral
dose: 450 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
CYSTEAMINE plasma
Homo sapiens
population: UNHEALTHY
age: CHILD
sex: FEMALE / MALE
food status: UNKNOWN
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
351 mg × min/L
404 mg/m² 4 times / day steady-state, oral
dose: 404 mg/m²
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
CYSTEAMINE plasma
Homo sapiens
population: UNHEALTHY
age: CHILD
sex: FEMALE / MALE
food status: UNKNOWN
96 μM × h
450 mg single, oral
dose: 450 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
CYSTEAMINE plasma
Homo sapiens
population: UNHEALTHY
age: CHILD
sex: FEMALE / MALE
food status: UNKNOWN
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
90 min
404 mg/m² 4 times / day steady-state, oral
dose: 404 mg/m²
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
CYSTEAMINE plasma
Homo sapiens
population: UNHEALTHY
age: CHILD
sex: FEMALE / MALE
food status: UNKNOWN
Funbound

Funbound

ValueDoseCo-administeredAnalytePopulation
48%
404 mg/m² 4 times / day steady-state, oral
dose: 404 mg/m²
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
CYSTEAMINE plasma
Homo sapiens
population: UNHEALTHY
age: CHILD
sex: FEMALE / MALE
food status: UNKNOWN
48%
450 mg single, oral
dose: 450 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
CYSTEAMINE plasma
Homo sapiens
population: UNHEALTHY
age: CHILD
sex: FEMALE / MALE
food status: UNKNOWN
Doses

Doses

DosePopulationAdverse events​
0.44 % 1 times / day multiple, ophthalmic
Recommended
Dose: 0.44 %, 1 times / day
Route: ophthalmic
Route: multiple
Dose: 0.44 %, 1 times / day
Sources: Page: 98 EI0109S
unhealthy, 13.4 (5.9-27.8)
n = 20
Health Status: unhealthy
Condition: cystinosis
Age Group: 13.4 (5.9-27.8)
Sex: M+F
Population Size: 20
Sources: Page: 98 EI0109S
Disc. AE: Intracranial hypertension...
AEs leading to
discontinuation/dose reduction:
Intracranial hypertension
Sources: Page: 98 EI0109S
600 mg 1 times / day multiple, oral
Recommended
Dose: 600 mg, 1 times / day
Route: oral
Route: multiple
Dose: 600 mg, 1 times / day
Sources: Page: RP103-05
healthy, 37.5 (19-64)
n = 20
Health Status: healthy
Age Group: 37.5 (19-64)
Sex: M+F
Population Size: 20
Sources: Page: RP103-05
Disc. AE: Blurred vision, Hematuria...
AEs leading to
discontinuation/dose reduction:
Blurred vision
Hematuria
Sources: Page: RP103-05
AEs

AEs

AESignificanceDosePopulation
Intracranial hypertension Disc. AE
0.44 % 1 times / day multiple, ophthalmic
Recommended
Dose: 0.44 %, 1 times / day
Route: ophthalmic
Route: multiple
Dose: 0.44 %, 1 times / day
Sources: Page: 98 EI0109S
unhealthy, 13.4 (5.9-27.8)
n = 20
Health Status: unhealthy
Condition: cystinosis
Age Group: 13.4 (5.9-27.8)
Sex: M+F
Population Size: 20
Sources: Page: 98 EI0109S
Blurred vision Disc. AE
600 mg 1 times / day multiple, oral
Recommended
Dose: 600 mg, 1 times / day
Route: oral
Route: multiple
Dose: 600 mg, 1 times / day
Sources: Page: RP103-05
healthy, 37.5 (19-64)
n = 20
Health Status: healthy
Age Group: 37.5 (19-64)
Sex: M+F
Population Size: 20
Sources: Page: RP103-05
Hematuria Disc. AE
600 mg 1 times / day multiple, oral
Recommended
Dose: 600 mg, 1 times / day
Route: oral
Route: multiple
Dose: 600 mg, 1 times / day
Sources: Page: RP103-05
healthy, 37.5 (19-64)
n = 20
Health Status: healthy
Age Group: 37.5 (19-64)
Sex: M+F
Population Size: 20
Sources: Page: RP103-05
OverviewDrug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
inconclusive
inconclusive
inconclusive
no
no
no
no
no
no
no
no
no
no
no
no
no
no
no
weak [Inhibition 780 uM]
weak [Inhibition 780 uM]
Drug as victim

Drug as victim

TargetModalityActivityMetaboliteClinical evidence
no
no
no
no
no
no
no
no
yes
yes
yes
yes
yes
yes
yes
yes
yes
PubMed

PubMed

TitleDatePubMed
Role of heme oxygenase-1 in the regulation of manganese superoxide dismutase gene expression in oxidatively-challenged astroglia.
2000 Oct
Pantetheinase activity of membrane-bound Vanin-1: lack of free cysteamine in tissues of Vanin-1 deficient mice.
2000 Oct 20
Oxidative stress, glucose-6-phosphate dehydrogenase and the red cell.
2001
Structure-activity analysis of the potentiation by aminothiols of the chromosome-damaging effect of bleomycin in G0 human lymphocytes.
2001
Sugar-mediated crosslinking of alpha-biotinylated-Lys to cysteamine-agarose support: a method to isolate Maillard Lys-Lys-like crosslinks.
2001 Apr
Comparison of different protein immobilization methods on quartz crystal microbalance surface in flow injection immunoassay.
2001 Dec 15
Pharmacological properties of a newly synthesized H(+)/K(+) ATPase inhibitor, 1-(2-methyl-4-methoxyphenyl)-4-.
2001 Jan 5
Gene expression and gene therapy in experimental duodenal ulceration.
2001 Jan-Dec
Therapy effect of antiulcer agents on new chronic cysteamine colon lesion in rat.
2001 Jan-Dec
Attenuation of radiation-induced genomic instability by free radical scavengers and cellular proliferation.
2001 Jul 1
Effect of centrally administered oxytocin on gastric and duodenal ulcers in rats.
2001 Jun
[Urease inhibition by polymer analogs of substrate and thiophosphamides].
2001 Mar-Apr
Potentiation of NMDA receptor function through somatostatin release: a possible mechanism for the cognition-enhancing activity of GABA(B) receptor antagonists.
2001 Sep
Role of porphyrin sequestration in the biogenesis of iron-laden astrocytic inclusions in primary culture.
2002
Effect of sperm survival and CTC staining pattern on in vitro fertilization of porcine oocytes.
2002 Apr 15
Cysteine-mediated electron transfer in syntrophic acetate oxidation by cocultures of Geobacter sulfurreducens and Wolinella succinogenes.
2002 Jul
Synthesis of (+/-)-madindolines and chemical models. Studies of chemical reactivity.
2002 Jul 11
Cysteamine supplementation during in vitro maturation and embryo culture: a useful tool for increasing the efficiency of bovine in vitro embryo production.
2002 Jun
Negligible urinary cysteamine loss in cystinosis patients with Fanconi syndrome.
2002 May
Characterization of the surfactin synthetase C-terminal thioesterase domain as a cyclic depsipeptide synthase.
2002 Nov 12
Identification and quantitation of key aroma compounds formed in Maillard-type reactions of fructose with cysteamine or isothiaproline (1,3-thiazolidine-2-carboxylic acid).
2002 Sep 11
Patents

Sample Use Guides

The recommended maintenance dose of 1.30 grams/m2/day. Patients over age 12 and over 110 pounds should receive 2.0 grams/day given in four divided doses as a starting maintenance dose. This dose should be reached after 4 to 6 weeks of incremental dosage increases as stated above. The dose should be raised if the leukocyte cystine level remains > 2 nmol/1⁄2 cystine/mg/protein.
Route of Administration: Oral
Oocytes were obtained from 4-6 weeks old Naval Medical Research Institute (NMRI) female mice, 48 hours after stimulation with Intraperitoneal (IP) injection of 10 IU Pregnant Mare Serum Gonadotropin (PMSG). Germinal Vesicle (GV) oocyte with and without cumulus cells were isolated from ovaries and cultured in Tissue Culture Medium (TCM) 199 with availability of 100 μM of CYS (Cysteamine). After 24 hours, mature oocyte in metaphase II (MII) were fertilized with sperm in In vitro Fertilization (IVF) medium (T6) and evaluated for fetal development into blastocyst. CYS could significantly (p<0.05) increase the rate of IVM and oocyte evolution, and embryo formation in medium culture.
Substance Class Chemical
Created
by admin
on Wed Jul 05 23:32:15 UTC 2023
Edited
by admin
on Wed Jul 05 23:32:15 UTC 2023
Record UNII
5UX2SD1KE2
Record Status Validated (UNII)
Record Version
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Name Type Language
CYSTEAMINE
HSDB   MI   USAN   VANDF  
USAN  
Official Name English
ETHANETHIOL, 2-AMINO-
Systematic Name English
CYSTEAMINE [MI]
Common Name English
CYSTEAMINE [USAN]
Common Name English
NSC-647528
Code English
CYSTEAMINE [INCI]
Common Name English
CYSTEAMINE [VANDF]
Common Name English
MERCAPTAMINE
INN   MART.   WHO-DD  
INN  
Official Name English
MEA
Common Name English
mercaptamine [INN]
Common Name English
MERCAPTAMINE [MART.]
Common Name English
2-Aminoethanethiol
Systematic Name English
L-1573
Code English
CYSTEAMINE [HSDB]
Common Name English
2-MERCAPTOETHYLAMINE
Systematic Name English
Mercaptamine [WHO-DD]
Common Name English
Classification Tree Code System Code
LIVERTOX NBK548701
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
WHO-ATC S01XA21
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
FDA ORPHAN DRUG 231006
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
NCI_THESAURUS C29701
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
NDF-RT N0000175544
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
WHO-VATC QS01XA21
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
FDA ORPHAN DRUG 404613
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
WHO-ATC A16AA04
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
FDA ORPHAN DRUG 13986
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
EU-Orphan Drug EU/3/11/928
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
FDA ORPHAN DRUG 55690
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
FDA ORPHAN DRUG 262408
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
FDA ORPHAN DRUG 258008
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
NDF-RT N0000187051
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
NDF-RT N0000175549
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
WHO-VATC QA16AA04
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
FDA ORPHAN DRUG 368712
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
Code System Code Type Description
SMS_ID
100000085466
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
RXCUI
3022
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY RxNorm
NSC
647528
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
EVMPD
SUB08770MIG
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
NCI_THESAURUS
C61696
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
WIKIPEDIA
CYSTEAMINE
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
HSDB
7353
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
DRUG BANK
DB00847
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
CHEBI
17141
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
INN
195
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
DRUG CENTRAL
768
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
MESH
D003543
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
ECHA (EC/EINECS)
200-463-0
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
ChEMBL
CHEMBL602
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
PUBCHEM
6058
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
DAILYMED
5UX2SD1KE2
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
IUPHAR
7440
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
EPA CompTox
DTXSID3022875
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
CAS
60-23-1
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
FDA UNII
5UX2SD1KE2
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
USAN
CC-44
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
CHEBI
58029
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY
MERCK INDEX
M4046
Created by admin on Wed Jul 05 23:32:15 UTC 2023 , Edited by admin on Wed Jul 05 23:32:15 UTC 2023
PRIMARY Merck Index
Related Record Type Details
TARGET -> INHIBITOR
BINDER->LIGAND
BINDING
SALT/SOLVATE -> PARENT
SALT/SOLVATE -> PARENT
SALT/SOLVATE -> PARENT
Related Record Type Details
PRODRUG -> METABOLITE ACTIVE
Related Record Type Details
ACTIVE MOIETY
Name Property Type Amount Referenced Substance Defining Parameters References
Volume of Distribution PHARMACOKINETIC