Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients

Cystinosis is a severe inherited metabolic storage disease caused by the lysosomal accumulation of cystine. Lifelong therapy with drug cysteamine bitartrate necessary. Cysteamine cleaves intralysosomal cystine, and thereafter, it can exit from organelle. The need for frequent dosing every 6 h high prevalence gastrointestinal side effects lead to poor adherence. purpose our study was improve treatment comparing efficacy two formulas. This highly relevant long-term outcome cystinosis patients. cystine levels 17 patients taking immediate-release (IR-cysteamine/Cystagon®) encapsulated delayed-release (EC-cysteamine) were analyzed. EC-cysteamine showed near-ideal pharmacokinetic profile indicative delayed release (longer Tmax Tmin), corresponding few fluctuations. In addition, Cmax IR-cysteamine greater, which responsible unbearable (e.g., nausea, vomiting, halitosis, lethargy). Treatment improves quality life because frequency intake be reduced 2–3 times daily has more favorable than IR-cysteamine. particular, no access only approved Procysbi® could benefit cost-effective alternative.