The US Food and Drug Administration (FDA) has cleared Sigma-Tau Pharmaceuticals' topical ophthalmic therapeutic to treat corneal cystine crystals in cystinosis patients.
The CYSTARAN 0.44% solution, which is developed in collaboration with the National Institutes of Health (NIH), is designated as an orphan drug in the US.
The FDA approval was based on the clinical safety and efficacy data of CYSTARAN in controlled 300 patients clinical trials conducted by the NIH.
Results demonstrate the use of ophthalmic cysteamine as an effective treatment of corneal cystine crystals.
NIH National Human Genome Research Institute clinical director William Gahl said the FDA approval of the drug represents the culmination of collaboration among the National Eye Institute, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Human Genome Research Institute and Sigma-Tau Pharmaceuticals.
"It also has involved invaluable cooperation from cystinosis advocacy groups--the Cystinosis Research Network, the Cystinosis Foundation and the Cystinosis Research Foundation," Gahl added.
Sigma-Tau chief operating officer Dave Lemus said, "This new medicine will offer physicians the only FDA-approved treatment for patients with corneal cystine crystal accumulation, many of whom are children and whose lives are seriously impacted by this debilitating chronic condition."