Cysteamine solution receives FDA nod for treatment of cystinosisAugust 29, 2020 0 By FM
Cysteamine ophthalmic solution (0.37%) (Cystadrops) has recently been approved by the US FDA for the treatment of ocular manifestations of cystinosis, announced the company Recordati Rare Diseases.
Cystinosis is a complex rare genetic disorder that causes an accumulation of the amino acid cystine within cells, forming crystals that can build up and damage the cells. These crystals negatively affect many systems in the body, especially the kidneys and eyes.
Cysteamine solution is a viscous eye drop that reduces corneal cystine crystal deposits in patients with cystinosis. The solution is recommended to be applied four times a day, according to the agency.
According to a phase 3 trial of cysteamine, 15 patients treated with the formulation experienced a 40% reduction in corneal cystine crystal density as assessed by in vivo confocal microscopy from baseline to 90 days.
The most common adverse effects of the drops were stinging, blurred vision, eye irritation, eye redness, application site discomfort, eye itching, watery eyes and medicine deposit around the eyes.