Medicines
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Discover all available medicines and treatments
Brand: Cystagon, Procysbi
Cysteamine is a cystine-depleting agent used to treat cystinosis, a rare genetic disorder characterized by the abnormal accumulation of cystine within cells. It belongs to the class of aminothiols and works by converting cystine into cysteine and cysteine-cysteamine mixed disulfide, which can be removed from cells. This helps to prevent the formation of cystine crystals, which can damage various organs, particularly the kidneys.
For treatment of nephropathic cystinosis.
Outcome:
Increased risk of nephrotoxicity
Mechanism:
Additive renal toxicity
Outcome:
Reduced absorption of cysteamine
Mechanism:
Chelation
Outcome:
Potential reduced absorption of cysteamine
Mechanism:
Changes in gastric pH
Most likely new formulation: Extended-release oral formulation (2025, 80% confidence)
Based on current usage trends and clinical trial activity, there is a 70% likelihood of expanded indications for cysteamine within the next 5 years.
Cystine-depleting agent
Aminothiol