The FDA approves the first drug, cenegermin, for the treatment of neurotrophic keratitis, a rare disease affecting the cornea.
The US Food and Drug Administration (FDA) has approved Dompé farmaceutici SpA’s cenegermin (Oxervate) for the treatment of a rare disease affecting the cornea, neurotrophic keratitis; this is the first drug approved to combat this degenerative disease.
“While the prevalence of neurotrophic keratitis is low, the impact of this serious condition on an individual patient can be devastating,” Wiley Chambers, MD, an ophthalmologist in the FDA’s Center for Drug Evaluation and Research, said in a recent statement. “In the past, it has often been necessary to turn to surgical interventions; these treatments are usually the only palliative in this disease.”
The approval marks a major advancement in the fight against the disease and offers complete corneal healing for many of the patients affected by it, Dr. Chambers added.
Two randomized, controlled, multicenter, double-masked studies demonstrated the safety and efficacy of the topical eye drop containing cenegermin in 151 patients with the disease.
For the first study, participants with the disease in only 1 eye were randomized into 3 different treatment groups: one group received Oxervate, a second group received an eye drop containing a different concentration of cenegermin, and the third group received an eye drop without cenegermin. For all treatment groups, eye drops were administered 6 times, on a daily basis, for the duration of 8 weeks.
For the second study, patients with the disease in both eyes were randomized into 2 treatment groups: one treated with Oxervate eye drops and the other group treated with eye drops that did not contain cenegermin. As in the first study, all treatment groups received eye drops, bilaterally, 6 times daily, for the duration of 8 weeks.
For both studies, investigators reported complete corneal healing in 8 weeks in 70% of the patients treated with the Oxervate eye drops as compared with only 28% of patients who were treated with eye drops that did not contain cenegermin.
Eye pain, ocular hyperemia, eye inflammation, and increased lacrimation, are listed as the most common adverse reactions experienced by patients who received the Oxervate eye drops.
Previously, the Oxervate eye drops were granted priority review and orphan drug designations.