Investigational Drug Details
| Drug ID: | D095 |
| Drug Name: | Cysteamine bitartrate |
| Synonyms: | 2-amino-1-ethanethiol; 2-amino-ethanethiol; 2-aminoethanethiol; beta-aminoethanethiol; beta-Mercaptoethylamine; Cysteamine; MEA; Mercaptamine; Thioethanolamine; β-aminoethylthiol; β-MEA |
| Type: | Chemical drug |
| DrugBank ID: | DB00847 |
| DrugBank Description: | Cystinosis is a rare disease caused by mutations in the CTNS gene that encodes for cystinosin, a protein responsible for transporting cystine out of the cell lysosome. A defect in cystinosin function is followed by cystine accumulation throughout the body, especially the eyes and kidneys. Several preparations of cysteamine exist for the treatment of cystinosis manifestations, some in capsule form, and others in ophthalmic solution form. In particular, cystine deposits on the eye can cause significant discomfort throughout the day and require frequent treatment with eye drops, typically every waking hour. On August 25th 2020, the first ophthalmic solution for cystinosis requiring only 4 daily treatments was granted FDA approval. Cysteamine eye drops are a practical and effective option for those affected by ocular cystinosis. Marketed by Recordati Rare Diseases Inc., CYSTADROPS® reduce the burden of multiple frequent medications normally administered to those with cystinosis. |
| PubChem ID: | 23111531 |
| CasNo: | 27761-19-9 |
| Repositioning for NAFLD: | Yes |
| SMILES: | C(CS)N.[C@@H]([C@H](C(=O)O)O)(C(=O)O)O |
| Structure: |
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| InChiKey: | NSKJTUFFDRENDM-ZVGUSBNCSA-N |
| Molecular Weight: | 227.238 |
| DrugBank Targets: | Cystine cleavage; Neuropeptide Y receptor type 2 other/unknown |
| DrugBank MoA: | Individuals born without the ability to metabolize cystine suffer from cystinosis, a rare genetic disorder characterized by the widespread accumulation of cystine crystals throughout the body and eye tissues. The cystine crystals may cause considerable damage, particularly in the renal tissues and corneal tissues. In some cases, renal failure can occur during childhood if the condition is left untreated. Other organs that may be affected by cystinosis include the CNS, thyroid, pancreas, muscle tissues, and gonads. Cysteamine converts cystine to cysteine and cysteine-cysteamine mixed disulfides, reducing the buildup of corneal cystine crystals. This drug participates in a thiol-disulfide interchange reaction with lysosomes, leading to cysteine exit from the lysosome in patients diagnosed with cystinosis. |
| DrugBank Pharmacology: | Cystine accumulation is the cause of organ damage in cystinosis. Cysteamine prevents the accumulation of cystine crystals in the body and is specifically prescribed to prevent kidney and eye damage. Cysteamine converts cystine into a form that may easily exit cells, preventing harmful accumulation. |
| DrugBank Indication: | The bitartrate salt of cysteamine is used for the oral treatment of nephropathic cystinosis and cystinuria in children 6 years old and above, and adults. The hydrochloride salt, used in eye drop preparations, is indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis. |
| Targets: | -- |
| Therapeutic Category: | -- |
| Clinical Trial Progress: | Phase 2&3 completed (NCT01529268: In a randomized trial, we found that 1 year of CBDR did not reduce overall histologic markers of NAFLD compared with placebo in children. Children receiving CBDR, however, had significant reductions in serum aminotransferase levels and lobular inflammation.) |
| Latest Progress: | Under clinical trials |
| Trial ID | Source ID | Phases | Status | Study Results | Start Date | Last Update Posted | |
|---|---|---|---|---|---|---|---|
| L0944 | NCT01529268 | Phase 2/Phase 3 | Not recruiting | Has Results | 18/01/2012 | 16 December 2017 | Details |
| Strategy ID | Strategy | Synonyms | Related Targets | Related Drugs |
|---|
| Article ID | PMID | Source | Title | |
|---|---|---|---|---|
| A06984 | 32673798 | Mol Metab | Non-alcoholic fatty liver disease and steatohepatitis: State of the art on effective therapeutics based on the gold standard method for diagnosis. | Details |
| A07625 | 32416645 | Hepatology | Alanine Aminotransferase and Gamma-Glutamyl Transpeptidase Predict Histologic Improvement in Pediatric Nonalcoholic Steatohepatitis. | Details |
| A13822 | 29740791 | Paediatr Drugs | Nonalcoholic Fatty Liver Disease in Children: Not a Small Matter. | Details |
| A15013 | 29128056 | Clin Liver Dis | Emerging Treatments for Nonalcoholic Fatty Liver Disease and Nonalcoholic Steatohepatitis. | Details |
| A15239 | 29028128 | Hepatology | Diagnostic accuracy of magnetic resonance imaging hepatic proton density fat fraction in pediatric nonalcoholic fatty liver disease. | Details |
| A17436 | 27569726 | Gastroenterology | In Children With Nonalcoholic Fatty Liver Disease, Cysteamine Bitartrate Delayed Release Improves Liver Enzymes but Does Not Reduce Disease Activity Scores. | Details |
| A21508 | 25267322 | Aliment Pharmacol Ther | Review article: the management of paediatric nonalcoholic fatty liver disease. | Details |
| A24212 | 23113697 | Fundam Clin Pharmacol | Pharmacokinetics of cysteamine bitartrate following intraduodenal delivery. | Details |
| A24667 | 22633783 | J Pediatr | The effect of cysteamine bitartrate on adiponectin multimerization in non-alcoholic fatty liver disease and healthy subjects. | Details |
| A25850 | 21395631 | Aliment Pharmacol Ther | Enteric-coated cysteamine for the treatment of paediatric non-alcoholic fatty liver disease. | Details |