FDA Approves First Drug for Neurotrophic Keratitis

Orphan Drug May Provide Complete Corneal Healing in Some Patients

The FDA has approved Oxervate (cenegermin, Dompé farmaceutici SpA), a topical eye drop that is the first drug for the treatment of neurotrophic keratitis. This rare degenerative disease results from a loss of corneal sensation, which impairs corneal health and causes progressive damage to the top layer of the cornea, resulting in corneal thinning, ulceration, and perforation, in severe cases. The prevalence of neurotrophic keratitis has been estimated to be less than five in 10,000 individuals.

The safety and efficacy of Oxervate were studied in a total of 151 patients with neurotrophic keratitis in two eight-week, randomized controlled multi-center, double-blinded studies. In the first study, patients were randomized into three groups. 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. In the second study, patients were randomized into two groups. 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. 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 most common adverse reactions in patients taking Oxervate are eye pain, ocular hyperemia, eye inflammation, and increased lacrimation.

Oxervate was granted orphan drug designation.

Source: FDA, August 22, 2018