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

Details

Stereochemistry ABSOLUTE
Molecular Formula C4H6O6.C2H7NS
Molecular Weight 227.236
Optical Activity UNSPECIFIED
Defined Stereocenters 2 / 2
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of CYSTEAMINE BITARTRATE

SMILES

NCCS.O[C@H]([C@@H](O)C(O)=O)C(O)=O

InChI

InChIKey=NSKJTUFFDRENDM-ZVGUSBNCSA-N
InChI=1S/C4H6O6.C2H7NS/c5-1(3(7)8)2(6)4(9)10;3-1-2-4/h1-2,5-6H,(H,7,8)(H,9,10);4H,1-3H2/t1-,2-;/m1./s1

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

Molecular Formula C4H6O6
Molecular Weight 150.0868
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 2 / 2
E/Z Centers 0
Optical Activity UNSPECIFIED

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

1994
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
Cholesterol 7alpha-hydroxylase. 2. Biochemical properties and participation of endogenous cholesterol in the assay in vitro.
1975 Jun 16
Taurine and hypotaurine in spermatozoa and epididymal fluid of cats.
2001
Oxidative stress, glucose-6-phosphate dehydrogenase and the red cell.
2001
Synthesis and anti-melanoma activity of analogues of N-acetyl-4-S-cysteaminylphenol substituted with two methyl groups alpha to the nitrogen.
2001 Feb
Adsorption properties and activities of lipase on a gold substrate modified by self-assembled monolayers.
2001 Nov
Gastric antisecretory and antiulcer activity of oxytocin in rats and guinea pigs.
2001 Nov 21
Role of porphyrin sequestration in the biogenesis of iron-laden astrocytic inclusions in primary culture.
2002
Role of heme oxygenase-1 in the biogenesis of corpora amylacea.
2002
A novel strategy for the synthesis of neoglycoconjugates from deacylated deep rough lipopolysaccharides.
2002
In vitro maturation and glutathione synthesis of porcine oocytes in the presence or absence of cysteamine under different oxygen tensions: role of cumulus cells.
2002
Interventions for paracetamol (acetaminophen) overdoses.
2002
Enhancement of solar inactivation of Escherichia coli by titanium dioxide photocatalytic oxidation.
2002
Electrochemical and spectroelectrochemical behavior of the main photodegradation product of nifedipine: the nitrosopyridine derivative.
2002 Apr
Modulation of inflammatory paw oedema by cysteamine in the rat.
2002 Apr
Effects of tea from Turnera ulmifolia L. on mouse gastric mucosa support the Turneraceae as a new source of antiulcerogenic drugs.
2002 Apr
Generation of roasted notes based on 2-acetyl-2-thiazoline and its precursor, 2-(1-hydroxyethyl)-4,5-dihydrothiazole, by combined bio and thermal approaches.
2002 Apr 10
Effect of sperm survival and CTC staining pattern on in vitro fertilization of porcine oocytes.
2002 Apr 15
The ability of hippocampal CA1 area for induction of long-term potentiation is persistently reduced by prior treatment with cysteamine: an in vitro study.
2002 Aug
Differentiating the orientations of photosynthetic reaction centers on Au electrodes linked by different bifunctional reagents.
2002 Aug
In vivo confocal microscopy of the cornea in nephropathic cystinosis.
2002 Dec
Glutathione in gingival crevicular fluid and its relation to local antioxidant capacity in periodontal health and disease.
2002 Dec
Lysosomal cystine storage augments apoptosis in cultured human fibroblasts and renal tubular epithelial cells.
2002 Dec
Evidence for a central inhibitory role of growth hormone secretagogues and ghrelin on gastric acid secretion in conscious rats.
2002 Feb
Generating favorable nano-environments for thermal and solvent stabilization of immobilized beta-galactosidase.
2002 Feb 15
A molecular CT blood pool contrast agent.
2002 Jul
Cysteine-mediated electron transfer in syntrophic acetate oxidation by cocultures of Geobacter sulfurreducens and Wolinella succinogenes.
2002 Jul
Cystinosis.
2002 Jul 11
Synthesis of (+/-)-madindolines and chemical models. Studies of chemical reactivity.
2002 Jul 11
Gastroprotective effect of adrenomedullin administered subcutaneously in the rat.
2002 Jun
X-ray crystallographic studies on butyryl-ACP reveal flexibility of the structure around a putative acyl chain binding site.
2002 Jun
Cysteamine supplementation during in vitro maturation and embryo culture: a useful tool for increasing the efficiency of bovine in vitro embryo production.
2002 Jun
The effects of lysosomotropic agents on normal and INCL cells provide further evidence for the lysosomal nature of palmitoyl-protein thioesterase function.
2002 Jun 13
Age-related prevalence of anterior segment complications in patients with infantile nephropathic cystinosis.
2002 Mar
Prevalence of apoptosis and inner cell allocation in bovine embryos cultured under different oxygen tensions with or without cysteine addition.
2002 Mar 15
Effect of glutathione synthesis stimulation during in vitro maturation of ovine oocytes on embryo development and intracellular peroxide content.
2002 Mar 15
Polymer-cysteamine conjugates: new mucoadhesive excipients for drug delivery?
2002 Mar 2
Subunit modification and association in VR1 ion channels.
2002 Mar 22
Negligible urinary cysteamine loss in cystinosis patients with Fanconi syndrome.
2002 May
Properties of selected S-nitrosothiols compared to nitrosylated WR-1065.
2002 May
Endogenous somatostatin inhibits interaction of insulin and cholecystokinin on exocrine secretion of isolated, perfused rat pancreas.
2002 May
Tumour targeting of humanised cross-linked divalent-Fab' antibody fragments: a clinical phase I/II study.
2002 May 6
Characterization of the surfactin synthetase C-terminal thioesterase domain as a cyclic depsipeptide synthase.
2002 Nov 12
Cysteamine pre-treatment reduces pentylenetetrazol-induced plasticity and epileptiform discharge in the CA1 region of rat hippocampal slices.
2002 Nov 15
Nephropathic cystinosis associated with cardiomyopathy: a 27-year clinical follow-up.
2002 Nov 9
Follow-up and treatment of adults with cystinosis in the Netherlands.
2002 Oct
Possible mechanism for involvement of cysteine in aroma production in wine.
2002 Oct 9
Studies on the anti-inflammatory and anti-nociceptive effects of melatonin in the rat.
2002 Sep
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
Structural determination and odor characterization of N-(2-mercaptoethyl)-1,3-thiazolidine, a new intense popcorn-like-smelling odorant.
2002 Sep 11
Electric potential control of DNA immobilization on gold electrode.
2003 Jan
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 Fri Dec 15 15:58:57 GMT 2023
Edited
by admin
on Fri Dec 15 15:58:57 GMT 2023
Record UNII
QO84GZ3TST
Record Status Validated (UNII)
Record Version
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Name Type Language
CYSTEAMINE BITARTRATE
MI   ORANGE BOOK   VANDF  
Common Name English
MERCAMINE BITARTRATE
Common Name English
CYSTEAMINE BITARTRATE [ORANGE BOOK]
Common Name English
Mercaptamine bitartrate [WHO-DD]
Common Name English
CYSTAGON
Brand Name English
CYSTEAMINE BITARTRATE [JAN]
Common Name English
ETHANETHIOL, 2-AMINO-, (2R,3R)-2,3-DIHYDROXYBUTANEDIOATE (1:1)
Systematic Name English
MERCAPTAMINE BITARTRATE [MART.]
Common Name English
RP-103
Code English
.BETA.-MERCAPTOETHYLAMINE HYDROGEN TARTRATE
Systematic Name English
CYSTEAMINE BITARTRATE [MI]
Common Name English
MERCAPTOETHYLAMINE BITARTRATE
Systematic Name English
MERCAPTAMINE BITARTRATE
MART.   WHO-DD  
Common Name English
CYSTEAMINE BITARTRATE [VANDF]
Common Name English
PROCYSBI
Brand Name English
Classification Tree Code System Code
NCI_THESAURUS C29701
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
EU-Orphan Drug EU/3/14/1306
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
Code System Code Type Description
CHEBI
50386
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
DAILYMED
QO84GZ3TST
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
ChEMBL
CHEMBL602
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
CAS
27761-19-9
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
SMS_ID
100000089423
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
RXCUI
81990
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY RxNorm
ECHA (EC/EINECS)
248-641-7
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
DRUG BANK
DBSALT000032
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
EPA CompTox
DTXSID801026168
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
MERCK INDEX
m4046
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY Merck Index
FDA UNII
QO84GZ3TST
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
NCI_THESAURUS
C47467
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
EVMPD
SUB03168MIG
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
PUBCHEM
11958147
Created by admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
PRIMARY
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