First FDA Approval for Rare Eye Disease Neurotrophic Keratitis

The US Food and Drug Administration approved cenegermin (Oxervate), for the treatment of neurotrophic keratitis, a rare disease affecting the cornea. This is the first approved drug for this rare eye condition is estimated to affect fewer than 5 in 10,000 individuals.

“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 the past, it has often been necessary to turn to surgical interventions; these treatments are usually only palliative in this disease.”

Patients with neurotrophic keratitis experience the loss of corneal sensation, which leads to progressive damage to the top layer of the cornea. This damage can include corneal thinning and ulceration, and, in severe cases, perforation of the cornea.

Results from 2 randomized, controlled, double-masked studies of eye drops containing cenegermin contributed to the FDA’s approval decision. The studies included a total of 151 patients with neurotrophic keratitis.

In the first study, patients were randomized into 3 groups that received: Oxervate eye drops, eye drops with another concentration of cenegermin, or placebo eye drops. The second study randomized patients to either Oxervate or placebo eye drops. All drops were given 6 times daily for 8 weeks.

Together, the studies found that 70% of patients receiving Oxervate achieved complete corneal healing in 8 weeks, compared to 28% of patients treated with placebo eye drops.

“Today’s approval provides a novel topical treatment and a major advance that offers complete corneal healing for many of these patients,” Chambers said.

The most commonly reported adverse reactions included eye pain, ocular hyperemia (enlarged blood vessels in the white of the eyes), eye inflammation and increased lacrimation (watery eyes).

The FDA previously granted Priority Review and Orphan Drug designation to Oxervate.