CYSTEAMINE

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 Search

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
Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2007/020392s010lbl.pdf
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.

CNS Activity

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Protein-glutamine gamma-glutamyltransferase 2 7 Target ID: P21980 Gene ID: 7052.0 Gene Symbol: TGM2 Target Organism: Homo sapiens (Human) Sources: https://www.ncbi.nlm.nih.gov/pubmed/15675041	Inhibitor 8228		178.0 µM [IC50]

Conditions

Condition	Modality	Targets	Highest Phase	Product
Nephropathic cystinosis 6 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2007/020392s010lbl.pdf	Primary		Approved 8360	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

C_max

Value	Dose	Co-administered	Analyte	Population
2.7 mg/L DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213491s000lbl.pdf	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 DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2013/203389s000lbl.pdf	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

Value	Dose	Co-administered	Analyte	Population
351 mg × min/L DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213491s000lbl.pdf	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 DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2013/203389s000lbl.pdf	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

T_1/2

Value	Dose	Co-administered	Analyte	Population
90 min DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213491s000lbl.pdf	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

F_unbound

Value	Dose	Co-administered	Analyte	Population
48% DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213491s000lbl.pdf	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% DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2013/203389s000lbl.pdf	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

Dose	Population	Adverse 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2012/200740Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2012/200740Orig1s000MedR.pdf Page: 98 EI0109S	Disc. AE: Intracranial hypertension... AEs leading to discontinuation/dose reduction: Intracranial hypertension Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2012/200740Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000MedR.pdf Page: RP103-05	Disc. AE: Blurred vision, Hematuria... AEs leading to discontinuation/dose reduction: Blurred vision Hematuria Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000MedR.pdf Page: RP103-05

AEs

AE	Significance	Dose	Population
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2012/200740Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2012/200740Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000MedR.pdf 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000MedR.pdf Page: RP103-05

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
substrate 1709 inhibitor 1579 inducer 768	substrate 1010 inhibitor 1752	substrate 1193 inhibitor 1837

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP1A2 1509 CYP2B6 799 CYP2C8 901 CYP2C19 1463 CYP2E1 876 CYP2A6 704 P-gp 1437 BCRP 691 OATP1B1 781 OATP1B3 700 OAT1 595 OAT3 636 OCT1 506 OCT2 638	CYP1A2 1032 CYP2B6 660 CYP2C8 688 CYP2C19 985 CYP2E1 648 CYP2A6 523 P-gp 1527 BCRP 555 OATP1B1 403 OATP1B3 347 OAT1 320 OAT3 333 OCT1 322 OCT2 348	CYP1A2 689 CYP2B6 538

Drug as perpetrator

Target	Modality	Activity
CYP1A2 1673	inducer 1542 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	inconclusive
CYP2B6 985	inducer 1542 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	inconclusive
CYP3A4 1909	inducer 1542 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	inconclusive
BCRP 765	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	no
CYP1A2 1673	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP2A6 736	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP2B6 985	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP2C19 1537	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP2C8 955	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP2C9 1803	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP2D6 1875	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP2E1 964	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
CYP3A4 1909	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=20 Page: 20.0	no
OAT1 599	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	no
OAT3 638	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	no
OATP1B1 796	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	no
OATP1B3 709	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	no
P-gp 1626	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	no
OCT1 523	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	weak [Inhibition 780 uM]
OCT2 639	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	weak [Inhibition 780 uM]

Drug as victim

Target	Modality	Activity
BCRP 555	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	no
CYP2A6 523	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	no
CYP3A4 1709	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	no
OAT1 320	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=23 Page: 23.0	no
OAT3 333	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=23 Page: 23.0	no
OATP1B1 403	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=23 Page: 23.0	no
OATP1B3 347	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=23 Page: 23.0	no
OCT1 322	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=23 Page: 23.0	no
CYP1A2 1032	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	yes
CYP2B6 660	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	yes
CYP2C19 985	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	yes
CYP2C8 688	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	yes
CYP2C9 1010	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	yes
CYP2D6 1193	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	yes
CYP2E1 648	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=16 Page: 16.0	yes
OCT2 348	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=23 Page: 23.0	yes
P-gp 1527	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000ClinPharmR.pdf#page=22 Page: 22.0	yes

PubMed

Title	Date	PubMed
Role of heme oxygenase-1 in the regulation of manganese superoxide dismutase gene expression in oxidatively-challenged astroglia.	2000 Oct	10942521
Pantetheinase activity of membrane-bound Vanin-1: lack of free cysteamine in tissues of Vanin-1 deficient mice.	2000 Oct 20	11042271
Oxidative stress, glucose-6-phosphate dehydrogenase and the red cell.	2001	11765001
Structure-activity analysis of the potentiation by aminothiols of the chromosome-damaging effect of bleomycin in G0 human lymphocytes.	2001	11246218
Sugar-mediated crosslinking of alpha-biotinylated-Lys to cysteamine-agarose support: a method to isolate Maillard Lys-Lys-like crosslinks.	2001 Apr	11393357
Comparison of different protein immobilization methods on quartz crystal microbalance surface in flow injection immunoassay.	2001 Dec 15	11730334
Pharmacological properties of a newly synthesized H(+)/K(+) ATPase inhibitor, 1-(2-methyl-4-methoxyphenyl)-4-.	2001 Jan 5	11137875
Gene expression and gene therapy in experimental duodenal ulceration.	2001 Jan-Dec	11595457
Therapy effect of antiulcer agents on new chronic cysteamine colon lesion in rat.	2001 Jan-Dec	11595451
Attenuation of radiation-induced genomic instability by free radical scavengers and cellular proliferation.	2001 Jul 1	11425485
Effect of centrally administered oxytocin on gastric and duodenal ulcers in rats.	2001 Jun	11747752
[Urease inhibition by polymer analogs of substrate and thiophosphamides].	2001 Mar-Apr	11357424
Potentiation of NMDA receptor function through somatostatin release: a possible mechanism for the cognition-enhancing activity of GABA(B) receptor antagonists.	2001 Sep	11522321
Role of porphyrin sequestration in the biogenesis of iron-laden astrocytic inclusions in primary culture.	2002	12401955
Effect of sperm survival and CTC staining pattern on in vitro fertilization of porcine oocytes.	2002 Apr 15	12041695
Cysteine-mediated electron transfer in syntrophic acetate oxidation by cocultures of Geobacter sulfurreducens and Wolinella succinogenes.	2002 Jul	12070769
Synthesis of (+/-)-madindolines and chemical models. Studies of chemical reactivity.	2002 Jul 11	12098241
Cysteamine supplementation during in vitro maturation and embryo culture: a useful tool for increasing the efficiency of bovine in vitro embryo production.	2002 Jun	11984830
Negligible urinary cysteamine loss in cystinosis patients with Fanconi syndrome.	2002 May	12036193
Characterization of the surfactin synthetase C-terminal thioesterase domain as a cyclic depsipeptide synthase.	2002 Nov 12	12416979
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	12207481

Patents

Sample Use Guides

In Vivo Use Guide

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

In Vitro Use Guide

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

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

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