Cenegermin for the Treatment of Moderate or Severe Neurotrophic Keratopathy: Results from a Prospective, Phase IV, Open-Label Study in China

INTRODUCTION: Neurotrophic keratopathy (NK) is a rare degenerative disease that can lead to epithelial breakdown, corneal ulceration, and potentially perforation and vision loss. Cenegermin is a recombinant human nerve growth factor approved for stage 2/3 NK, though data on its efficacy outside of United States/European populations are limited. METHODS: This prospective, phase IV, open-label, multicenter study investigated the efficacy, safety, and pharmacokinetics (PK) of cenegermin eye drops in Chinese patients with stage 2/3 NK in a routine clinical setting (CTR20220066). Patients received cenegermin eye drops (20 mcg/mL; 6 times/day at 2-hour intervals) for 8 weeks with follow-up to week 56. The primary endpoint was corneal healing at week 8 per investigator assessment. Additional endpoints included lesion worsening, corneal healing through week 56, safety, and PK. RESULTS: Of the 28 patients receiving cenegermin, 22/26 (84.6%, 95% confidence interval 65.1-95.6%; 2 missing) achieved corneal healing at week 8; 3/22 (13.6%) reported recurrence during 48-week follow-up but 20/22 (90.9%) were healed at week 56; and 2/28 (7.1%) experienced lesion worsening during 8-week treatment. In addition, 25/28 (89.3%) experienced ≥ 1 treatment-emergent adverse event (TEAE); for the majority (23/28, 82.2%) of these patients, TEAEs were mild/moderate. None of the serious TEAEs reported by 10 (35.7%) patients were assessed as related to cenegermin or led to treatment discontinuation. Eye pain was the most common cenegermin-related TEAE (5/28, 17.9%), primarily limited to the treatment period. CONCLUSIONS: These findings demonstrate that cenegermin eye drops are an effective, generally well-tolerated treatment in Chinese patients with stage 2/3 NK, supporting their use in this patient population. TRIAL REGISTRATION: CTR20220066.