The FDA just greenlighted Italian firm Dompé’s eye drop Oxervate for the treatment of neurotrophic keratitis (NK), a rare type of eye disease. While the condition may sound unfamiliar, the approval is special in that it marks three firsts in the U.S. drug history.
After winning orphan drug and priority review designations, Oxervate on Wednesday became the first FDA-approved drug for NK, which is estimated to affect fewer than 65,000 persons in the U.S.
The degenerative disease triggers a loss of corneal sensation, which can eventually result in vision loss if left untreated. Before Oxervate, available treatments were either palliative or, in the case of surgeries, invasive.
Oxervate is a recombinant human nerve growth factor (NGF) that resembles the human body’s natural NGF. It works directly on corneal epithelial cells to stimulate their growth and recovery. As an eye drop, Oxervate is the first application of a human NGF as drug and the first topical biologic approved in ophthalmology.
“By directly promoting corneal healing, Oxervate has the potential to change the way neurotrophic keratitis is treated, and may eventually result in a new standard of care for patients with this rare condition,” said Reza Dana, professor of ophthalmology at Harvard Medical School and Dompé’s medical adviser, in a statement.
In two clinical studies on a total of 151 subjects, Oxervate completely healed the cornea in 70% of patients after eight weeks, compared with 28% among those who received placebo eye drops. Among those healed by Oxervate, about 80% remained so after one year.
The FDA nod came a year after Oxervate won a go-ahead in Europe, and it's also a first for Dompé. According to Dompé’s chairman Sergio Dompé, Oxervate is the company’s first therapy to be sold in the U.S.
It now plans to roll out the drug in early 2019, through a commercial operation it has in place in the San Francisco Bay Area, a media aide for the company told FiercePharma. As for pricing, the company “is speaking with insurers and providers in the U.S. to make sure that they price Oxervate in a way that makes it available to patients, and also reflects its first-in-class nature and potential to completely heal NK,” she said.
Editor's Note: The story has been updated with Dompé's commercial and pricing plan for Oxervate.