FDA approves Dompé’s Oxervate for rare eye disease treatment

Dompé farmaceutici, an Italian pharmaceutical company, has achieved a milestone with the U.S. Food and Drug Administration (FDA) approval of Oxervate (cenegermin) eye drops for the treatment of neurotrophic keratitis, a rare degenerative eye disease. This approval marks Oxervate as the first FDA-sanctioned drug specifically for this condition.

Details on Neurotrophic Keratitis:

Neurotrophic keratitis is characterized by a loss of corneal sensation that leads to progressive damage to the cornea, including thinning, perforation, and ulceration in severe cases. The FDA notes that although neurotrophic keratitis affects less than five in 10,000 people, its impact on those affected can be severe and debilitating.

Clinical Trials and Efficacy:

The approval of Oxervate was supported by the results of two eight-week double-masked safety and efficacy trials involving 151 patients with neurotrophic keratitis. The studies demonstrated that Oxervate could achieve complete corneal healing within eight weeks in 70% of the patients, a significant improvement over the 28% healing rate observed in patients treated without cenegermin.

Expert Commentary:

Dr. Wiley Chambers, an ophthalmologist at the FDA’s Center for Drug Evaluation and Research, commented on the approval, stating, “While the prevalence of neurotrophic keratitis is low, the impact of this serious condition on an individual patient can be devastating. Today’s approval provides a novel topical treatment and a major advance that offers complete corneal healing for many of these patients.”

Adverse Reactions and Safety Profile:

The most common adverse reactions reported during the trials were eye pain, ocular hyperemia (enlarged blood vessels in the white area of the eyes), eye inflammation, and increased lacrimation (watery eyes).

Prior Designations and European Approval:

Oxervate had previously received Orphan Drug and Priority Review designations by the FDA, underscoring its significance in treating a rare and serious condition. Additionally, the drug was approved by the European Commission in July 2017 for the treatment of moderate to severe neurotrophic keratitis.

The FDA approval of Oxervate is a significant advancement in the treatment of neurotrophic keratitis, providing a new and effective option for patients suffering from this rare condition. By offering a treatment that can achieve complete corneal healing, Oxervate not only improves the quality of life for patients but also reduces the need for more invasive treatments like surgery. This approval highlights the importance of targeted therapies in managing and potentially reversing the effects of rare and severe diseases.