FDA Approves First Drug for Neurotrophic Keratitis
Officials with the FDA have approved a topical eye drop containing cenegermin, the first drug indicated for the treatment of a rare disease affecting the cornea.
Officials with the FDA have approved a topical eye drop containing cenegermin (Oxervate, Dompé farmaceutici SpA). The product is the first drug permitted by the agency for the treatment of neurotrophic keratitis, a rare disease affecting the cornea.
“While the prevalence of neurotrophic keratitis is low, the impact of this serious condition on an individual patient can be devastating,” said Wiley Chambers, MD, an ophthalmologist in the FDA’s Center for Drug Evaluation and Research, in a prepared statement. “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.”
Neurotrophic keratitis is a degenerative disease resulting from a loss of corneal sensation, which causes progressive damage to the top layer of the cornea. Corneal impairment due to loss of corneal sensation includes corneal thinning, ulceration, and perforation in severe cases. The prevalence of neurotrophic keratitis has been estimated to be less than 5 in 10,000 individuals.
According to the FDA, the safety and efficacy of Oxervate was studied in a total of 151 patients with neurotrophic keratitis in 2, 8-week, randomized controlled multicenter, double-masked studies. In the first study, patients were randomized into 3 different 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 2 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 6 times daily in the affected eye(s) for 8 weeks.
In the first study, only patients with the disease in 1 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 8 weeks was demonstrated in 70% of patients treated with Oxervate compared to 28% of patients treated without cenegermin.
The most common adverse reactions in patients taking Oxervate are eye pain, enlarged blood vessels in the white of the eyes, eye inflammation, and increased watery eyes, according to the FDA.
Dompé farmaceutici SpA was granted Priority Review designation for Oxervate. The FDA’s goal with this designation is to take action on an application within 6 months of the application being filed. If approved, the drug is determined by the agency that it would provide a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition. Oxervate also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
FDA approves first drug for neurotrophic keratitis, a rare eye disease [news release]. Silver Spring, MD; August 22, 2018: FDA website. http://www.pharmacytimes.com/link/210. Accessed August 23, 2018.