Study update: Cenegermin for moderate or severe neurotrophic keratopathy

Recently reported findings indicate that cenegermin-bkbj ophthalmic solution (Oxervate; Dompé) may perform as an effective and generally well-tolerated option for patients in China with moderate or severe neurotrophic keratopathy (NK).¹ Cenegermin is a recombinant human nerve growth factor approved for stage 2/3 NK.

This prospective, phase IV, open-label, multicenter study, conducted by Prof. Dr. Weiyun Shi and colleagues, assessed the efficacy, safety, and pharmacokinetics (PK) of cenegermin in Chinese patients with unilateral stage 2 (moderate) or stage 3 (severe) NK, using the Mackie classification and evidence of decreased corneal sensitivity (less than or equal to 4 cm using the Cochet-Bonnet esthesiometer), fulfilling the label authorization requirements to be treated with cenegermin in China.¹

Patients were treated with 1 drop of cenegermin (20 mcg/mL) in the conjunctival sac of the affected eye(s) 6 times daily at 2-hour intervals for a duration of 8 weeks. Ocular and safety assessments were conducted at weeks 2, 4, 6, and 8 during treatment, followed by an additional 48-week follow-up period. No other topical ocular therapies were allowed throughout the treatment phase.¹

The primary efficacy end point of the study was the number of patients who achieved complete healing of the persistent corneal epithelial defect (PED) or corneal ulcer by the end of the 8-week treatment period.¹ A number of secondary end points were also evaluated.

By week 8, 22 out of 26 patients had achieved complete healing of the PED or corneal ulcer, although most showed signs of corneal recovery earlier in the treatment course. The median time to corneal healing was 15.5 days. Lesion worsening, as assessed by the investigator, occurred in 2 patients, and 3 patients experienced a recurrence of the PED or corneal ulcer during the follow-up period.¹

Mean (SD) best-corrected distance visual acuity (ETDRS letters) increased from 17.0 at baseline to 42.4 at week 8 (n = 26) and further improved to 49.9 at week 56 (n = 27). The mean (SD) change from baseline was 25.4 letters at week 8 and 33.4 letters at week 56.¹

The occurrence and severity of treatment-emergent adverse events (TEAEs), treatment-related TEAEs, and serious TEAEs were monitored throughout the 56-week study. In addition, intraocular pressure measurements, slit lamp examinations, fundus ophthalmoscopy, and external ocular assessments were conducted at scheduled timepoints during the study.

A total of 28 patients met the eligibility criteria and received at least one dose of the study medication, making them part of the efficacy and safety analysis set. One patient withdrew because of a TEAE of mild eye pain, which was deemed related to the study drug but not serious. Two patients were excluded from the primary end point analysis due to early treatment discontinuation and the absence of an assessment, respectively.¹

The authors noted treatment-related TEAEs as “rare,” occurring in 14.3% of patients during treatment and 3.7% during follow-up.¹ Eye pain was the most frequently reported treatment-related TEAE in both periods. No serious TEAEs were observed during the treatment phase.

The authors concluded that the findings of this study “support and expand upon those of previous randomized clinical trials, demonstrating the efficacy and safety of cenegermin for the treatment of moderate-to-severe NK.” They suggest that cenegermin is an effective and well-tolerated therapy for Chinese patients with stage 2/3 NK, reinforcing its use in this population.¹

Cenegermin progress advances as Dompé gains FDA national priority voucher

In other updates, Dompé was selected by the FDA for the Commissioner’s National Priority Voucher program, gaining a shortened review timeline and enhanced collaboration.^2,3 Announced in June 2025, the program reduces review times to 1 to 2 months, though extensions may occur, according to the company.

Dompé noted it plans to use the voucher for a Biologic License Application for intranasal cenegermin-bkbj to treat nonarteritic anterior ischemic optic neuropathy.² A registrational trial is planned across more than 130 sites in 16 countries, the company noted. Nine vouchers were issued, with cenegermin-bkbj the only eye care product, according to Dompé. Additional recipients will be announced in the future.

References

1. Li S, Xu J, Yuan J, et al. Cenegermin for the treatment of moderate or severe neurotrophic keratopathy: results from a prospective, phase IV, open-label study in China. Ophthalmol Ther. 2025;14(12):3021-3033. doi:10.1007/s40123-025-01253-5

2. Dompé's program in NAION selected for landmark FDA Commissioner’s National Priority Voucher Program. News release. Published October 16, 2025. Accessed October 20, 2025. https://www.dompe.com/us/media/press-releases/dompe-s-program-in-naion-selected-for-landmark-fda-commissioner-s-national-priority-voucher-program/

3. FDA awards first-ever national priority vouchers to nine sponsors. News release. Published October 16, 2025. Accessed October 20, 2025. https://www.fda.gov/news-events/press-announcements/fda-awards-first-ever-national-priority-vouchers-nine-sponsors