Dompé Receives FDA Approval of Oxervate, the First Drug for Neurotrophic Keratitis

Source: Dompé

The FDA has approved the first drug to treat neurotrophic keratitis, a rare and progressive eye disease that can lead to corneal scarring and vision loss.

Oxervate (cenegermin-bkbj ophthalmic solution), which is manufactured by Italian pharmaceutical company Dompé, 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.

“This is Dompé’s first approval in the United States, and our first time in the [US] market,” Nathalie Dompé, Vice President, Business Development at Dompé Pharmaceuticals, said in an interview with Eyewire News. “The approval was based on two randomized, double-masked trials. And up to three-quarters of the patients were healed after an 8-week cycle. And of these, up to 80% of patients were completely healed after 1 year, and 95% did not experience another epithelial defect after 1 year, which I think is very impressive data.”

“Neurotrophic keratitis can be disabling, hard to treat, and many patients do not respond well to existing therapies,” Reza Dana, Professor of Ophthalmology at Harvard Medical School, Director of the Cornea Service, Senior Scientist at the Massachusetts Eye and Ear, and Dompé medical advisor, said in a company news release. “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.”

Neurotrophic keratitis is a rare orphan condition that affects fewer than 65,000 persons in the United States based on estimated disease prevalence. It results from impaired function of corneal nerves, which can be caused by herpetic or other infections, ocular surface injuries, ocular or neurologic surgeries, and some systemic conditions that can impair corneal sensation. If unchecked, the disease can progress in severity, leading to persistent epithelial defects, corneal ulcers, melting, perforation and vision loss. Until now, treatment options for neurotrophic keratitis were limited to symptomatic treatments, which do not target the underlying disease pathology. These include artificial tears, antibiotics, autologous serum-derived eye drops, tarsorrhaphy (a procedure in which the eyelids are partially sutured together) and botulinum-induced ptosis (closure of the eyelid). Other surgical interventions, designed to restore the integrity of the cornea, include conjunctival flap surgeries and corneal transplants, which are invasive and can compromise the appearance and function of the eye.1

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. The company’s subsequent trials demonstrated the safety and effectiveness of cenegermin-bkbj for the treatment of neurotrophic keratitis. Oxervate represents the first-ever topical biologic medication approved in ophthalmology, and is the first ever application of a human NGF as drug or treatment.

Oxervate is approved for use in adults and in children 2 years of age and older. Oxervate is taken over an 8-week period in which patients can easily self-administer the treatment at home. Investigated as a monotherapy, Oxervate offers patients a treatment option that could prevent the need for invasive procedures.

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