Attendees at this year’s Women in Ophthalmology meeting took part in a quick game of $1 or Mystery Box! In this edition, participants tested their knowledge of neurotrophic keratitis.
Neurotrophic keratitis (NK) is a rare, potentially sight-threatening degenerative corneal disease caused by impaired sensory innervation, which disrupts normal healing and can progress from epithelial breakdown to persistent defects, ulcers, or perforation.1 Risk factors include diabetes, shingles, herpes zoster, and preserved glaucoma medications.1
NK prevalence is estimated at 21.34 cases per 100,000 patients, translating to roughly 70,000 people in the U.S.1-3 True prevalence is likely underestimated due to undiagnosed early or mild cases, though earlier detection by ophthalmologists and optometrists is improving this.1
When testing corneal sensitivity, which eye first?
Can you name the only FDA-approved treatment for NK?
According to the Mackey classification, which stage of NK is shown in the image?
Why does catching it early actually matter?
NK exists on a continuum, and waiting for classic late-stage findings—stromal ulceration, corneal melt—means missing the window where treatment is most effective.
A 2025 cohort study found that most NK patients presented at moderate to severe stages, and these later presentations accounted for the majority of complications, including corneal scarring, thinning, secondary infection, and vision loss.4 Patients diagnosed and managed earlier, by contrast, showed better potential for corneal healing and visual recovery.4
Enter OXERVATE
OXERVATE (cenegermin-bkbj) remains the only FDA-approved recombinant human nerve growth factor (rhNGF) therapy for NK.5 Rather than simply supporting the ocular surface, it targets the underlying nerve damage by binding TrkA and p75NTR receptors throughout the cornea, promoting nerve regeneration and epithelial repair.5
This mechanism is part of why cenegermin is increasingly used earlier in the disease course: Phase 4 data from the DEFENDO trial support treating stage 1 NK, when restoring corneal sensation offers the best odds of preventing progression to ulceration or surgery.6
What's next for NK treatment?
Cenegermin was the first mechanism-targeted therapy for NK, but the pipeline behind it is growing. RGN-259, a topical thymosin beta-4 formulation, is now in a phase 3 trial for neurotrophic keratopathy, and BRM424, a regenerative peptide that stimulates limbal stem cell activity, holds FDA Orphan Drug Designation and is progressing through phase 2.7-8
As these and other candidates mature, clinicians will have more nerve-targeted options to match to disease stage—but the data so far all point to the same conclusion: earlier recognition remains the single biggest lever for protecting vision in NK.
