Ophthalmic Solution Granted FDA Approval for Treatment of Cystinosis


The cysteamine ophthalmic solution drops are applied 4 times a day.

The FDA has approved cysteamine ophthalmic solution 0.37% (Cystadrops, Recordati Rare Diseases, Inc.) for the treatment of patients living with cystinosis. The solution is the first FDA-approved cysteamine eye drop formulation that reduces corneal crystals with dosing 4 times a day, according to Andrea Recordati, CEO of Recordati Rare Diseases.

Cystinosis is a rare genetic condition present from birth. The condition causes the build-up of cystine crystals throughout the body, which causes widespread tissue and organ damage, and significantly impacts the eyes. Patients living with cystinosis often have sensitivity to light, eye discomfort and pain. Recent developments in treatment have led to increased life expectancies of those living with the disease.

In a phase 1/2a open label, controlled, randomized study across multiple centers, 15 patients were given cysteamine ophthalmic solution 0.37%. Eight participants also were part of an adaptive dose-responsive clinical trial. Corneal cystine density was assessed using in vivo confocal microscopy (IVCM)

The study found that patients receiving cysteamine ophthalmic solution 0.37% saw a 40% reduction in IVCM scores across all corneal layers 90 days after the baseline. Those in the adaptive dose-response clinical trial saw a 30% decrease in IVCM score, which was maintained through the 5-year study period.

“People living with cystinosis and their caregivers have to manage multiple medications every day. To reduce their daily burden, Recordati worked to develop a new viscous eye drop formulation for treating corneal cystine crystals,” said Recordati, in a prepared statement.

Common adverse effects associated with cysteamine ophthalmic solution was stinging, blurred vision, eye irritation, and eye redness. It is applied 4 times daily as a drop during waking hours and can be stored for up to 7 days at room temperature after opening.


U.S. FDA Approves CYSTADROPS® (Cysteamine Ophthalmic Solution) 0.37%, A New Practical Treatment Option for the Ocular Manifestations of Cystinosis (Press release) White Oak, MD, August 28, 2020 EurekAlert! Accessed August 28, 2020

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