The FDA has approved Oxervate for treatment of neurotrophic keratitis, a progressive disease affecting the cornea in fewer than five in 10,000 people, according to an Agency press release.
Oxervate (cenegermin), developed by Dompé farmaceutici SpA, is the first FDA approved drug for the rare disease, which can cause loss of corneal sensation and impair corneal health. Patients with neurotrophic keratitis can experience corneal thinning, ulceration, and perforation in severe cases.
Neurotrophic keratitis has been a “very frustrating disease for physicians,” Flavio Mantelli, MD, PhD, chief medical officer for Dompé, told Healio.com/OSN.
“It was called one of the most challenging conditions to treat. All physicians could do was protect the cornea as much as possible. They could provide constant lubrication with preservative-free artificial tears...but they could only slow down the progression, it was not a treatment. When the patients reached the end-stage of [neurotrophic keratitis], corneal melting or corneal perforation, the only option available was corneal surgery,” he said.
“Today’s approval provides a novel topical treatment and a major advance that offers complete corneal healing for many of these patients,” Wiley Chambers, MD, ophthalmologist at the FDA Center for Drug Evaluation and Research, said in the release.
The safety and efficacy of the topical eye drop was studied in 151 patients with neurotrophic keratitis in two 8-week, randomized, controlled, multi-center, double-masked studies. In both studies, patients were given the drops six times daily in the affected eyes for 8 weeks. Across both studies, 70% of patients treated with Oxervate experienced complete corneal healing in 8 weeks compared with 28% of patients who were not treated with the active ingredient, cenegermin, according to the release.
Eye pain, ocular hyperemia, eye inflammation and increased lacrimation were the most common adverse events in patients taking the drops.
The definitive results of the studies and the effectiveness of the drug in treating both European and U.S. populations make Oxervate a “breakthrough” for the treatment of neurotrophic keratitis, Mantelli said.
“It’s very important for patients because it changes the natural history of their condition. For the clinicians, it changes the management of their patients from chronic management to an 8-week treatment cycle. It’s quite unique,” he said.
Mantelli noted the drug could be commercialized in the United States by January 2019. – by Robert Linnehan
Disclosure: Mantelli reports he is the chief medical officer for Dompé farmaceutici SpA