Mercaptamine hydrochloride for treating corneal cystine crystal deposits in children aged 6 months to less than 2 years with cystinosis

Mercaptamine hydrochloride is being developed for the treatment of corneal cystine crystal deposits in children aged 6 months to 2 years with cystinosis. Cystinosis is a rare inherited disease in which excess amounts of cystine, an amino acid (protein building-block) naturally found in the body, build up within cells, especially in the kidneys and the eyes, damaging them. In corneal crystal accumulation, cystine crystals are deposited in the cornea (the transparent front layer of the eye). This creates vision problems like sensitivity to light, soreness and blurry vision which could slowly progress to visual impairment as the patient grows older. Starting treatment early can make a big difference in how well children with cystinosis who have an accumulation of cystine in their eyes do over time. Mercaptamine hydrochloride is currently approved for use in adults and children aged 2 years and older, there is a need for a suitable treatment option for cystinosis with corneal crystal accumulation in children aged 6 months to less than 2 years.