Abstract
Cystadrops® (viscous cysteamine hydrochloride 0.55% eye-drops solution) is the first orphan drug approved in the EU for the treatment of corneal crystal deposits in adults and children aged >2 years with cystinosis. The solution reduced the accumulation of cystine crystals in the cornea and the severity of photophobia in patients of all ages with cystinosis in clinical studies. Instillation adverse effects (e.g. burning and stinging) may occur, but generally are very transient and mild to moderate in severity. Relative to extemporaneously prepared formulations of cysteamine hydrochloride eye drops, Cystadrops® requires less frequent administration (relieving treatment burden), displays better stability (making storage, distribution and use by patients less difficult) and provides reliable standardization and availability.
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