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Introduction <p>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.</p> Methods <p>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&#xa0;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.</p> Results <p>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.</p> Conclusions <p>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.</p> Trial Registration <p>CTR20220066.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年10月5日

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  • Suxia Li,
  • Jianjiang Xu,
  • Jin Yuan,
  • Zhiqiang Pan,
  • Giacomo Siri,
  • Scott G. Hauswirth,
  • Flavio Mantelli,
  • Weiyun Shi

摘要

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.