The first drug, Oxervate (cenegermin) has been approved for the treatment of neurotrophic keratitis by the U.S. Food and Drug Administration. The prevalence of neurotrophic keratitis has been estimated to be less than five in 10,000 individuals. It is a rare disease degenerative disease resulting from a loss of corneal sensation.
This loss of corneal sensation leads to progressive damage to the top layer of the cornea, including corneal thinning, ulceration, and perforation in severe cases.
“While the prevalence of neurotrophic keratitis is low, the impact of this serious condition on an individual patient can be devastating,” said Wiley Chambers, M.D., an ophthalmologist in the FDA’s Center for Drug Evaluation and Research. “In the past, it has often been necessary to turn to surgical interventions; these treatments are usually only palliative in this disease. Today’s approval provides a novel topical treatment and a major advance that offers complete corneal healing for many of these patients.”
Oxervate, is a topical eye drop containing cenegermin whose safety and efficacy was studied in a total of 151 patients with neurotrophic keratitis in two, eight-week, randomized controlled multi-center, double-masked studies.
The patients were randomized into three different groups in the first study. While one group received Oxervate, a second group received an eye drop with a different concentration of cenegermin, and the third group received an eye drop without cenegermin.
The patients were randomized into two groups in the second study. One group was treated with Oxervate eye drops and the other group was treated with an eye drop without cenegermin. All eye drops in both studies were given six times daily in the affected eye(s) for eight weeks. In the first study, only patients with the disease in one eye were enrolled, while in the second study, patients with the disease in both eyes were treated in both eyes (bilaterally). Across both studies, complete corneal healing in eight weeks was demonstrated in 70 percent of patients treated with Oxervate compared to 28 percent of patients treated without cenegermin (the active ingredient in Oxervate).
The most common adverse reactions observed in patients taking Oxervate are eye pain, ocular hyperemia (enlarged blood vessels in the white of the eyes), eye inflammation and increased lacrimation.
Oxervate was granted Priority Review designation and it also received Orphan Drug designation earlier, which provides incentives to assist and encourage the development of drugs for rare diseases.
Dompé farmaceutici SpA has been granted approval of Oxervate by the FDA.