FDA Approves First Drug for Rare Eye Disease

August 24, 2018

Dompé Farmaceutici’s Oxervate (cenegermin), a topical eye drop, is the first treatment approved by the agency for neurotrophic keratitis, a rare disease affecting the cornea.

On Aug. 22, 2018, FDA approved Oxervate (cenegermin), a topical eye drop, the first treatment approved by the agency for neurotrophic keratitis, a rare disease affecting the cornea, the clear layer that covers the colored portion of the front of the eye.

According to Dompé Farmaceutici, the Milan-based developer of the drug,Oxervate is based on cenegermin-bkbj, a novel recombinant human nerve growth factor (rhNGF) that is structurally identical to the nerve growth factor (NGF) protein that is made in the human body, including in the ocular tissues. The endogenous protein supports corneal integrity though several mechanisms. NGF acts directly on corneal epithelial cells to stimulate their growth and survival. In addition, NGF is known to bind receptors on lacrimal glands to promote tear production, which may provide the eye with lubrication and natural protection from pathogens and injury. The protein also has been shown experimentally to support corneal innervation, which is lost in neurotrophic keratitis.

Neurotrophic keratitis is a degenerative disease resulting from a loss of corneal sensation. The loss of corneal sensation impairs corneal health causing progressive damage to the top layer of the cornea, including corneal thinning, ulceration, and perforation in severe cases. The prevalence of neurotrophic keratitis has been estimated to be less than five in 10,000 individuals.

Dompé reports that the regenerative potential of NGF was discovered by Nobel-prize winning scientists, but its therapeutic potential was not realized in ophthalmology until Dompé’s research and development center in L’Aquila, Italy, created cenegermin-bkbj, a recombinant version of human NGF, through a unique development process. FDA granted Oxervate orphan drug, fast track status, and breakthrough therapy designations, as well as priority review.

In May 2017, the European Medicines Agency (EMA) granted Oxervate marketing authorization. According to EMA, the drug is under additional monitoring, meaning that it is being monitored more intensively than other medicines because it is new to the market or has limited data of long-term use.

“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 FDA’s Center for Drug Evaluation and Research, in an agency press release. “In the past, it has often been necessary to turn to surgical interventions; these treatments are usually only palliative in this disease. Today’s approval provides a novel topical treatment and a major advance that offers complete corneal healing for many of these patients.”

Source: FDADompé Farmaceutici