cysteamine

Trade name: cystagon

Small moleculeapprovedOrphan Drug ^FDA

Approved

Aug 15, 1994

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. — NCATS

Clinical trial activity

28 trials · 29 clinical orgs · 4 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started Jul 6, 1978 (NCT00359684)

Timeline

1980s

Jan 1, 1986
Earliest Phase 2 Sponsor(trial)

1990s

Jan 25, 1991
Orphan Drug Designation
Aug 15, 1994
Mylan — Earliest FDA Approval
Aug 15, 1994
Mylan — NDA Organization
Jan 1, 1998
Earliest Phase 1 Sponsor(trial)

2010s

Jan 1, 2010
Horizon Pharma — Earliest Phase 3 Sponsor(trial)
Oct 2, 2012
Leadiant Biosciences, Inc. — NDA Secondary Org
Apr 30, 2013
Horizon Pharma — NDA Secondary Org
Apr 30, 2013
Horizon Pharma — Marketing Organization

2020s

Aug 19, 2020
Recordati — Marketing Organization
Aug 19, 2020
Recordati — NDA Secondary Org

Indications

Approved for

Cystinosis

Studied for

Mechanism of action

CystineREDUCING AGENT
Cystine reducing agent

Approval history

approvedPriority reviewAug 15, 1994

Chemistry & pharmacology

Loading structure…

SMILES

NCCS

Mol. weight: 77.149 g/mol
Lipinski Ro5: Pass
Rule of 3: Yes
Chirality: Achiral Molecule
Inorganic: No
Polymer: No
Delivery: Oral
Availability: Prescription Only

Oral

Yes

Parenteral

Topical

Sources

Also known as

2-amino-1-ethanethiol
2-amino-1-ethanethiol
2-aminoethanethiol
2-aminoethanethiol
2-amino-ethanethiol
2-amino-ethanethiol
2-mercaptoethylamine
beta-aminoethanethiol
beta-aminoethanethiol
beta-aminoethylthiol
beta-aminoethylthiol
beta-mea
beta-mea
beta-mercaptoethylamine
beta-mercaptoethylamine
ci-9148
ci-9148
cystagon
cystagon
cystagon
cystagon
cystagon
cystaran
cystaran
cysteamine
cysteamine
cysteamine
cysteamine
cysteamine
cysteamine
cysteamine
cysteamine bitartrate
cysteamine bitartrate
cysteamine bitartrate
cysteamine bitartrate
cysteamine bitartrate
cysteamine hcl
cysteamine hcl
cysteamine hcl
cysteamine hydrochloride
cysteamine hydrochloride
cysteamine hydrochloride
cysteamine hydrochloride
l-1573
l-1573
mea
mea
mea
mercamine
mercaptamin
mercaptamina
mercaptamina
mercaptamine
mercaptamine
mercaptamine
mercaptamine
mercaptamine
mercaptamine
mercaptamine bitartrate
mercaptamine bitartrate
mercaptamine bitartrate
mercaptamine bitartrate
mercaptamine bitartrate
mercaptamine bitartrate
mercaptamine bitartrate
mercaptamine hydrochloride
mercaptaminum
mercaptaminum
mercaptoethylamine
procysbi
procysbi
procysbi
thioethanolamine
thioethanolamine
thioethanolamine
β-aminoethylthiol
β-aminoethylthiol
β-mea
β-mea

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT06990984	Phase 2	Oct 31, 2025	Thiogenesis Therapeutics, Inc., University of Pennsylvania
NCT05994534	Phase 1/Phase 2 (Phase 2)	Oct 29, 2023	Nacuity Pharmaceuticals, Inc.
NCT05656833	N/A	Oct 28, 2022	Solta Medical, UnionDerm
NCT05206318	N/A	Dec 10, 2021	Chang Gung University
NCT04125927	Phase 3	Nov 30, 2019	Recordati
NCT05969587	Phase 3	Nov 28, 2019	China Medical University
NCT03883984	Phase 1	Apr 1, 2019	University of Cincinnati
NCT03000348	Phase 2	Dec 1, 2016	Agility Clinical, Inc., NovaBiotics Ltd., PSR Group B.V.
NCT02473445	Phase 2	May 1, 2015	Horizon Pharma
NCT02212431	Phase 1/Phase 2 (Phase 2)	Aug 1, 2014	Cystic Fibrosis Trust, University of Aberdeen, University of Huddersfield
NCT02023866	Phase 2	Jun 1, 2014	Horizon Pharma
NCT01733316	Phase 3	Jan 1, 2013	Horizon Pharma
NCT01529268	Phase 2/Phase 3 (Phase 3)	Jun 1, 2012	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NCT02012114	N/A	Dec 31, 2011	University of Lyon
NCT01432561	N/A	Sep 1, 2011	Raptor Pharmaceuticals Corp., University of California, San Diego
NCT01197378	Phase 3	Aug 1, 2010	Horizon Pharma
NCT01000961	Phase 3	Jun 1, 2010	Horizon Pharma
NCT01139125	N/A	Sep 1, 2009	Augusta University
NCT00872729	Phase 1/Phase 2 (Phase 2)	May 1, 2009	Horizon Pharma
NCT00799578	Phase 1/Phase 2 (Phase 2)	Oct 1, 2008	Raptor Pharmaceuticals Corp., University of California, San Diego

Showing 20 of 28 trials

Page 1 / 2

Organizations

Research & Development (29)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Horizon Pharma	For profit	6	6	2	2009
National Eye Institute (NEI)	Government	2	2	2	1986
Raptor Pharmaceuticals Corp.	For profit	2	0	2	2008
University of California, San Diego	Academic/Hospital	2	2	2	2008
Agility Clinical, Inc.	For profit	1	0	1	2016
Augusta University	Academic/Hospital	1	1	1	2009
Chang Gung University	Academic/Hospital	1	1	1	2021
China Medical University	Academic/Hospital	1	1	1	2019
Cystic Fibrosis Trust	Academic/Hospital	1	0	1	2014
Food and Drug Administration	Government	1	1	1	1999
Icahn School of Medicine at Mount Sinai	Academic/Hospital	1	1	1	2008
Leadiant Biosciences, Inc.	For profit	1	0	1	1999
Mylan	For profit	1	1	1	1999
Nacuity Pharmaceuticals, Inc.	For profit	1	1	1	2023
National Human Genome Research Institute (NHGRI)	Government	1	1	1	1978
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Government	1	1	1	2012
National Institutes of Health (NIH)	Government	1	1	1	2001
NovaBiotics Ltd.	For profit	1	1	1	2016
PSR Group B.V.	For profit	1	0	1	2016
Recordati	For profit	1	1	1	2019

29 organizations

Page 1 / 2

Marketing (6)

Organization	Org type	Relationship	Date
Horizon Pharma	For profit	NDA2	Apr 30, 2013
Horizon Pharma	For profit	MKTG	Apr 30, 2013
Leadiant Biosciences, Inc.	For profit	NDA2	Oct 2, 2012
Mylan	For profit	NDA	Aug 15, 1994
Recordati	For profit	MKTG	Aug 19, 2020
Recordati	For profit	NDA2	Aug 19, 2020