Cystadrops Approved to Treat Corneal Cystine Crystal Deposits

August 25, 2020 – The U.S. FDA has approved Cystadrops® (cysteamine ophthalmic solution) 0.37%, manufactured by Recordati Rare Diseases, to treat corneal cystine crystal deposits in adults and children who have cystinosis.

Cystinosis is a rare genetic disorder that causes cystine, a type of amino acid, to form damaging crystals inside the body’s cells. The majority of patients develop cystine crystals in their cornea during the course of the disease. Over time, this can lead to issues such as photophobia (sensitivity to light), the sensation of having a foreign body in the eye, visual impairment, blepharospasm (abnormal contraction of the eyelid), and features of glaucoma.

In one clinical trial, Cystadrops delivered a 40% reduction from baseline in corneal cystine crystal density at 90 days of treatment. The drug reduced corneal cystine crystal density by 30% in a second study, with the reduction maintained for the study’s full five-year period. Recommended dosing is one drop in each eye four times a day during waking hours. Cystadrops must be refrigerated until opened for use, at which point the bottle should be stored at room temperature and discarded after seven days.

The product has launched at a wholesale acquisition cost (WAC) of $1,750 per 5mL bottle.