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Penicillamine (D1-W12)
Penicillamine is a chelating agent that binds to heavy metals and promotes their urinary excretion, reducing toxic metal accumulation in tissues.
Penicillamine is a chelating agent that binds to heavy metals and promotes their urinary excretion, reducing toxic metal accumulation in tissues. Used for Wilson's disease (copper accumulation disorder), Cystinuria (cystine stone prevention).
At a glance
| Generic name | Penicillamine (D1-W12) |
|---|---|
| Also known as | D-penicillamine |
| Sponsor | Orphalan |
| Drug class | Chelating agent |
| Target | Heavy metal ions (copper, mercury, lead); thiol-reactive compounds |
| Modality | Small molecule |
| Therapeutic area | Rare disease / Metabolic disorder |
| Phase | Phase 3 |
Mechanism of action
Penicillamine contains a thiol group that forms stable complexes with heavy metals such as copper, mercury, and lead, facilitating their removal from the body through the kidneys. It also has immunomodulatory properties and may reduce collagen cross-linking. The drug has been used historically for Wilson's disease, cystinuria, and rheumatoid arthritis, though its exact mechanism in some conditions remains incompletely understood.
Approved indications
- Wilson's disease (copper accumulation disorder)
- Cystinuria (cystine stone prevention)
Common side effects
- Proteinuria
- Rash
- Taste disturbance
- Bone marrow suppression
- Lupus-like syndrome
Key clinical trials
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |