cysteamine (cystagon) Report issue

Small molecule Orphan Drug FDA Approved FDA Priority Review FDA
AACT ChEMBL DrugBank Drug Central Drugs@FDA KEGG MeSH PubChem repoDB

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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

Chemistry

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Pharmacology

  • Mechanism of Action:
  • Multi-specific: Missing data
  • Black Box: No
  • Availability: Prescription Only
  • Delivery Methods: Topical
  • Pro Drug: No

Drug Pricing (per unit)

United States

$0.2002 - $399.1703
More Pricing Detail

Note: This drug pricing data is preliminary, incomplete, and may contain errors.

Alternative names

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

Organizations (26)

Research & Development (26)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (4)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (9)

Approved Indications (1)


 

Cystinosis

Experimental Indications - Clinical Trial Phases 1-4 (9)


 

Asthma (Phase 1)

Cystic Fibrosis (Phase 2)

Cystinosis ()

Fatty Liver (Phase 1/Phase 2)

HIV Infections (Phase 2)

Infant (Phase 4)

Melanosis (Phase 3)

Mitochondrial Diseases (Phase 2)

Non-alcoholic Fatty Liver Disease (Phase 2/Phase 3)

Overview

  • Highest Phase: Phase 4
  • # of Trials: 27
  • # of Trial Organizations: 26

Trials by Phase

Trial Phase Start Date Organizations Indications

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