Penicillamine
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Also known as CuprimineD mercaptovalineD penicillamineDepenDimethylcysteineDistamineNSC-758421NSC-81549PenicilaminaTrolovolD-pencillaminebetabeta-dimethyl cysteineD-penicillamineSID29215023SID144204450D(-)-penicillamineD-(-)-PenicillaminePENICILLAMINE (CUPRIMINE)
Summary
Penicillamine (CHEMBL1430) is an approved small-molecule chelator (ATC M01CC01); indicated across 9 conditions including rheumatoid arthritis and rheumatic disorder.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: M01CC01
- Indications: 9 conditions
- Clinical trials: 8
- Chemistry: 149.21 Da · C5H11NO2S
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1430 |
| Name | Penicillamine |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 5852 |
| ChEBI | CHEBI:7959 |
| ATC | M01CC01 |
| Molecular formula | C5H11NO2S |
| Molecular weight | 149.21 |
| InChIKey | VVNCNSJFMMFHPL-VKHMYHEASA-N |
SMILES: CC(C)([C@H](C(=O)O)N)S
IUPAC name: (2S)-2-amino-3-methyl-3-sulfanylbutanoic acid
ChEBI definition: An optically active form of penicillamine having D-configuration. Pharmaceutical form (L-form is toxic) of chelating agent used to treat heavy metal poisoning.
Pharmacological roles (ChEBI): chelator, antirheumatic drug, drug allergen, copper chelator.
Also known as: Cuprimine, D mercaptovaline, D penicillamine, Depen, Dimethylcysteine, Distamine, NSC-758421, NSC-81549, Penicilamina, Penicillamine, Trolovol, D-pencillamine
Parent form; salt/anhydrous children: CHEMBL1469737
Patent coverage: 15,828 distinct patent families (57,837 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: 6 (assay-derived). Sample: Succinyl-diaminopimelate desuccinylase, Microtubule-associated protein tau, Fructose-bisphosphate aldolase, 4’-phosphopantetheinyl transferase ffp, Prostaglandin G/H synthase 1, Aldehyde dehydrogenase 1A1.
Bioactivity
ChEMBL activities: 3 potent at pChembl ≥ 5 of 8 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| PTGS1 | 6.08 | AC50 | 839.3 | nM | CHEMBL_ACT_25204943 |
| PTGS1 | 6.04 | AC50 | 905.2 | nM | CHEMBL_ACT_25205876 |
| MAPT | 5.1 | Potency | 7943 | nM | CHEMBL_ACT_4525796 |
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
9 indications (7 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 |
|---|---|---|---|
| rheumatoid arthritis | 4 | MONDO:0008383 | EFO:0000685 |
| rheumatic disorder | 4 | MONDO:0005554 | EFO:0005755 |
| movement disorder | 4 | MONDO:0005395 | EFO:0004280 |
| spondylitis | 4 | MONDO:0003937 | MONDO:0003937 |
| cystinuria | 4 | MONDO:0009067 | MONDO:0009067 |
| Wilson disease | 4 | MONDO:0010200 | MONDO:0010200 |
| central nervous system neoplasm | 2 | MONDO:0006130 | EFO:1000158 |
| head and neck cancer | 2 | MONDO:0005627 | EFO:0006859 |
1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 8.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 3 |
| PHASE3 | 2 |
| PHASE2/PHASE3 | 1 |
| EARLY_PHASE1 | 1 |
| Not specified | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT07465718 | PHASE3 | NOT_YET_RECRUITING | Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson’s Disease Patients. |
| NCT00552630 | PHASE2/PHASE3 | WITHDRAWN | Penicillamine Chelation for Children With Lead Poisoning |
| NCT03539952 | PHASE3 | COMPLETED | Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson’s Disease |
| NCT00003751 | PHASE2 | COMPLETED | Penicillamine, Low Copper Diet, and Radiation Therapy in Treating Patients With Glioblastoma |
| NCT03106948 | PHASE2 | UNKNOWN | Delivery of Inhibitors of Lysyl Oxidase (LysoLox) on Serial Angioplasty and Time to Restenosis |
| NCT06103617 | PHASE2 | UNKNOWN | Safety and Superiority of Penicillamine in Radiosensitization of Recurrent Head and Neck Cancer |
| NCT03957720 | EARLY_PHASE1 | UNKNOWN | The Individual Therapy for Patients With Wilson’s Disease |
| NCT01374282 | Not specified | NO_LONGER_AVAILABLE | Post Marketing Surveillance Study of Cuprimine |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).