Drug class

Oral Cystinosis Therapy, Cystine Depleting Agents

Oral cystinosis therapy, cystine-depleting agents comprise a specialized therapeutic class primarily represented by systemic formulations of cysteamine, including immediate-release and delayed-release cysteamine bitartrate. Read more about Oral Cystinosis Therapy, Cystine Depleting Agents

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About Oral Cystinosis Therapy, Cystine Depleting Agents.

Oral cystinosis therapy, cystine-depleting agents comprise a specialized therapeutic class primarily represented by systemic formulations of cysteamine, including immediate-release and delayed-release cysteamine bitartrate.

These medications function intracellularly by entering lysosomes and undergoing a thiol-disulfide exchange reaction with accumulated cystine, cleaving it into cysteine and a cysteine-cysteamine mixed disulfide.

These resultant molecules readily exit lysosomes via alternative amino acid export pathways, effectively bypassing the defective cystinosin transporter.

Clinically, these oral agents are indicated for the lifelong management of nephropathic cystinosis to decrease systemic intracellular cystine accumulation, delay renal failure, and mitigate extrarenal organ complications.