Cysteamine
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Also known as CystagonL-1573MEAMercaptaminaMercaptamineNSC-647528SID11111480SID90341118SID144206643
Summary
Cysteamine (CHEMBL602) is an approved small-molecule radiation protective agent (ATC A16AA04); indicated across 8 conditions including eye disorder and pneumonia.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: A16AA04 (+1 more)
- Indications: 8 conditions
- Clinical trials: 15
- Chemistry: 77.15 Da · C2H7NS
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL602 |
| Name | Cysteamine |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 6058 |
| ChEBI | CHEBI:17141 |
| ATC | A16AA04, S01XA21 |
| Molecular formula | C2H7NS |
| Molecular weight | 77.15 |
| InChIKey | UFULAYFCSOUIOV-UHFFFAOYSA-N |
SMILES: C(CS)N
IUPAC name: 2-aminoethanethiol
ChEBI definition: An amine that consists of an ethane skeleton substituted with a thiol group at C-1 and an amino group at C-2.
Pharmacological roles (ChEBI): radiation protective agent, geroprotector.
Other ChEBI roles (chemical / environmental): human metabolite, mouse metabolite.
Also known as: Cystagon, Cysteamine, L-1573, MEA, Mercaptamina, Mercaptamine, NSC-647528, cysteamine, SID11111480, SID90341118, CYSTEAMINE, SID144206643
Parent form; salt/anhydrous children: CHEMBL1200577, CHEMBL1256137, CHEMBL2062263, CHEMBL3763421
Patent coverage: 20,636 distinct patent families (61,585 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Thyrotropin receptor, Cytochrome P450 3A4.
Bioactivity
ChEMBL activities: 2 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| CYP3A4 | 6 | Potency | 1000 | nM | CHEMBL_ACT_4949423 |
| CYP3A4 | 6 | Potency | 1000 | nM | CHEMBL_ACT_5018302 |
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
8 indications (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| eye disorder | 4 | MONDO:0005328 | EFO:0003966 |
| pneumonia | 3 | MONDO:0005249 | EFO:0003106 |
| influenza | 3 | MONDO:0005812 | EFO:0007328 |
| cystinosis | 3 | MONDO:0016239 | MONDO:0016239 |
| severe acute respiratory syndrome | 3 | MONDO:0005091 | MONDO:0100096 |
| HIV infectious disease | 2 | MONDO:0005109 | EFO:0000764 |
| cystic fibrosis | 2 | MONDO:0009061 | MONDO:0009061 |
| fatty liver disease | 1 | MONDO:0004790 | HP:0001397 |
Clinical trials
Total trials: 15.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 4 |
| Not specified | 4 |
| PHASE4 | 2 |
| PHASE3 | 2 |
| PHASE1/PHASE2 | 2 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT00028262 | PHASE4 | COMPLETED | Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis |
| NCT01173965 | PHASE4 | COMPLETED | Endometrial Ablation With Non-hysteroscopic Methods |
| NCT02735707 | PHASE3 | RECRUITING | Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community- Acquired Pneumonia |
| NCT04125927 | PHASE3 | COMPLETED | Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) |
| NCT00001213 | PHASE2 | COMPLETED | Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis |
| NCT00002110 | PHASE2 | COMPLETED | A Phase II/III Study of Cysteamine (Mercaptoethylamine) and Zidovudine for the Treatment of HIV Disease |
| NCT00799578 | PHASE1/PHASE2 | COMPLETED | A Preliminary Study to Evaluate Cysteamine Therapy in Human Subjects With Non-Alcoholic Steatohepatitis (NASH) |
| NCT02212431 | PHASE1/PHASE2 | COMPLETED | First Study of Oral Cysteamine in Cystic Fibrosis |
| NCT03000348 | PHASE2 | COMPLETED | A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations |
| NCT07477288 | PHASE2 | COMPLETED | Effectiveness of a Bilayering Serum and Cream Containing GABA, DMAE, Cysteamine, and Bakuchiol for Skin Whitening and Anti-Aging |
| NCT00001736 | PHASE1 | COMPLETED | New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis |
| NCT00359684 | Not specified | RECRUITING | Use of Cysteamine in the Treatment of Cystinosis |
| NCT02766855 | Not specified | COMPLETED | Efficacy of Topical Cysteamine in Nephropathic Cyctinosis |
| NCT05206318 | Not specified | UNKNOWN | Efficacy and Safety of Topical Cysteamine for Postinflammatory Hyperpigmentation |
| NCT05656833 | Not specified | UNKNOWN | Combination Topical Cysteamine and Fractional 1927nm Low-Powered Diode Laser for Treatment of Facial Melasma |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline, but PharmGKB curates 1 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).