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Penicillamine (W12-W60)

Orphalan · Phase 3 active Small molecule

Penicillamine is a chelating agent that binds to heavy metals and promotes their urinary excretion, while also modulating immune responses through effects on collagen cross-linking and T-cell function.

Penicillamine is a chelating agent that binds to heavy metals and promotes their urinary excretion, while also modulating immune responses through effects on collagen cross-linking and T-cell function. Used for Wilson's disease (copper accumulation disorder), Cystinuria (amino acid metabolism disorder), Rheumatoid arthritis (historical use).

At a glance

Generic namePenicillamine (W12-W60)
Also known asD-Penicillamine
SponsorOrphalan
Drug classHeavy metal chelator / immunomodulator
TargetCopper, heavy metals (non-specific chelation); collagen cross-linking inhibition
ModalitySmall molecule
Therapeutic areaRare disease / Metabolic disorder
PhasePhase 3

Mechanism of action

Penicillamine works primarily as a heavy metal chelator by forming stable complexes with copper, mercury, lead, and other metals, facilitating their removal from the body via urine. Additionally, it inhibits collagen cross-linking and has immunomodulatory properties that reduce inflammatory responses, making it useful in autoimmune and connective tissue disorders. The drug's mechanism in Wilson's disease involves reducing copper accumulation in tissues.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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