FDA Approves Dompé’s Rare Eye Disease Drug

US regulators have approved the first drug to treat neurotrophic keratitis, a rare degenerative disease that progressively affects the cornea, the clear layer at the front of the eye, eventually leading to blindness if left untreated.

In a double first, Dompé farmaceutici’s Oxervate (cenegermin) is the first-ever application of a human nerve growth factor as a drug or treatment and the first-ever topical biologic medication approved in ophthalmology.

Dompé says its Oxervate topical eye drops have a mechanism that targets the root pathology of the disease. The Food and Drug Administration’s (FDA) clearance marks the Italian company’s first approval in the US, where the therapy had previously received Orphan Drug Designation, Fast Tract Status and Breakthrough Therapy Designation, which led to a priority review. The European Medicines Agency authorized cenegermin in 2017.

Clinical trials demonstrated that complete corneal healing occurred in eight weeks in 70% of patients treated with Oxervate compared to 28% of those who did not receive the therapy. Prior to Oxervate, the available treatments were either invasive surgery or palliative care.

“Neurotrophic keratitis can be disabling, hard to treat, and many patients do not respond well to existing therapies,” said Reza Dana, professor of ophthalmology at Harvard Medical School, director of the Cornea Service, senior scientist at Massachusetts Eye and Ear and Dompé medical advisor. “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.”

The company is reportedly planning to make Oxervate available in the US in early 2019.