FDA Clears First Drug for Neurotrophic Keratitis

Megan Brooks


August 23, 2018

The US Food and Drug Administration (FDA) has approved cenegermin (Oxervate, Dompé Farmaceutici S.p.A.), the first drug to treat neurotrophic keratitis.

Neurotrophic keratitis is a rare degenerative disease involving loss of corneal sensation, which leads to progressive damage to the top layer of the cornea, including corneal thinning and ulceration, and, in severe cases, perforation of the cornea. Cenegermin is a recombinant form of human nerve growth factor that stimulates growth and survival of corneal epithelial cells. 

"While the prevalence of neurotrophic keratitis is low, the impact of this serious condition on an individual patient can be devastating. In the past, it has often been necessary to turn to surgical interventions; these treatments are usually only palliative in this disease," Wiley Chambers, MD, ophthalmologist in the FDA's Center for Drug Evaluation and Research, said in a statement.

Cenegermin topical eye drops provide a "novel topical treatment and a major advance that offers complete corneal healing for many of these patients," said Chambers.

The safety and efficacy of cenegermin were studied in two randomized, controlled, double-blind studies involving a total of 151 patients with neurotrophic keratitis. Cenegermin eye drops were given six times daily in the affected eye(s) for 8 weeks.

Across both studies, complete corneal healing in 8 weeks occurred in 70% of patients treated with cenegermin compared with 28% of patients treated with inactive eye drops.

The most common adverse reactions in patients using cenegermin eye drops are eye pain, ocular hyperemia, eye inflammation, and increased lacrimation.

Cenegermin received priority review and had orphan drug designation.

The European Commission approved Cenegermin eye drops for treatment of neurotrophic keratitis last summer, following a thumbs up the European Medicines Agency's Committee for Medicinal Products for Human Use, as reported by Medscape Medical News.   

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