| Drug Name |
Cysteamine bitartrate |
| Drug ID |
BADD_D00557 |
| 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.[A218721]
Several preparations of cysteamine exist for the treatment of cystinosis manifestations, some in capsule form, and others in ophthalmic solution form.[L15606,L15616] 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.[L15611]
On August 25th 2020, the first ophthalmic solution for cystinosis requiring only 4 daily treatments was granted FDA approval.[L15606] 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.[L15601] |
| Indications and Usage |
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.[L15616] The hydrochloride salt, used in eye drop preparations, is indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.[L15601,L15606]
|
| Marketing Status |
approved; investigational |
| ATC Code |
A16AA04; S01XA21 |
| DrugBank ID |
DB00847
|
| KEGG ID |
D10468
|
| MeSH ID |
D003543
|
| PubChem ID |
23111531
|
| TTD Drug ID |
D0VD6Q
|
| NDC Product Code |
12828-0068; 0378-9045; 75987-145; 75987-100; 46439-8762; 17373-1199; 75987-140; 71666-010; 0378-9040; 75987-101 |
| UNII |
QO84GZ3TST
|
| Synonyms |
Cysteamine | beta-Mercaptoethylamine | beta Mercaptoethylamine | Mercaptoethylamine | Mercamine | Mercaptamine | 2-Aminoethanethiol | 2 Aminoethanethiol | Cysteinamine | Cystagon | Cysteamine Bitartrate | Bitartrate, Cysteamine | Cysteamine Tartrate (1:1) | Cysteamine Tartrate | Tartrate, Cysteamine | Cysteamine Dihydrochloride | Dihydrochloride, Cysteamine | Cysteamine Hydrochloride | Hydrochloride, Cysteamine | Cysteamine Maleate (1:1) | Cysteamine Tosylate | Tosylate, Cysteamine | Cysteamine, 35S-Labeled | 35S-Labeled Cysteamine | Cysteamine, 35S Labeled | Becaptan | Cysteamine Hydrobromide | Hydrobromide, Cysteamine |
