Drug reference

Penicillamine

Antidote · Treatment for Cystinosis

AntidoteTreatment for Cystinosis

CDSCO approved

EXCRETION

—

not curated

INTERACTIONS

12 major

SEVERE in our sources

PREGNANCY

Manufacturer advises avoid—teratogenic and toxic in animal studies.

FDA category + note

Top interactionssee all 12 →

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Mechanism

Penicillamine (D-β,β-dimethylcysteine) is a chelating agent that forms stable, water-soluble complexes with copper, mercury, lead, and other heavy metals, promoting their urinary excretion. In Wilson disease, it mobilizes hepatic copper deposits. Its disease-modifying effect in rheumatoid arthritis operates through a separate mechanism involving suppression of T-helper cell function, inhibition of macrophage activity, and reduction of IgM rheumatoid factor — effects that take 2-3 months to manifest.

Indications

Corneal cystine crystal deposits in patients with cystinosis (specialist use only)

Dosing

Adult: Oral for cystinosis (patients 50 kg and above): Initially one-sixth to one-quarter of the expected maintenance dose, increased gradually over 4–6 weeks to avoid intolerance; maintenance 2 g daily in 4 divided doses.…
Max dose: 2 g daily (oral maintenance)

Contraindications

History of hypersensitivity to penicillamine

Side effects

Common

Oral ulcerationDecreased taste acuityAlteration in taste sensationDry eye (topical)Eye discomfort (topical)Eye disorders (topical)Vision blurred (topical)Appetite decreased (oral)Asthenia (oral)Breath odour (oral)Diarrhoea (oral)Drowsiness (oral)Encephalopathy (oral)Fever (oral)Gastroenteritis (oral)Gastrointestinal discomfort (oral)Headache (oral)Nausea (oral)Skin reactions (oral)Vomiting (oral)

Serious