Clinically Urgently Needed Drug! Cenegermin Ophthalmic Solution Approved in China for Neurotrophic Keratitis

On August 14, cenegermin eye drops were approved for marketing in China for the treatment of neurotrophic keratitis. Developed by Dompé Farmaceutici S.p.A., this drug is included in the "First Batch of Clinically Urgent Overseas Drugs."

Neurotrophic KeratitisNeurotrophic keratitis is a rare ocular disorder caused by herpes or other infections, ocular surface injury, ocular or neurosurgical procedures, and systemic diseases such as diabetes that impair corneal sensation and trophism. If not promptly diagnosed and managed, the condition progressively worsens, leading to persistent epithelial defects, corneal ulceration, stromal melting, perforation, and vision loss.

Cenegermin ophthalmic solution is a topical eye drop administered six times daily for 8 weeks to treat neurotrophic keratitis. On December 14, 2015, cenegermin was designated as an orphan drug in the European Union. It was approved in the EU in July 2017 and in the United States in August 2018, becoming the first FDA-approved therapy for neurotrophic keratitis.

Cenegermin is a novel recombinant human nerve growth factor (rhNGF) that is structurally identical to the nerve growth factor (NGF) protein naturally produced in the human body and present in ocular tissues. This endogenous protein provides robust support for corneal integrity through multiple mechanisms. NGF acts directly on corneal cells to stimulate their growth and survival. Furthermore, NGF is known to bind to receptors on the lacrimal glands, promoting tear secretion to lubricate the eyes and provide natural protection against pathogens and injury. This protein has been demonstrated to support corneal innervation, a neural function that is lost in neurotrophic keratitis.

The efficacy and safety of cenegermin have been demonstrated in two independent, double-blind, randomized, multicenter, controlled clinical trials. This program includes the largest cohort of patients with neurotrophic keratitis enrolled in randomized controlled trials to date. Both studies evaluated monotherapy with cenegermin ophthalmic solution (20 μg/mL) in patients with moderate or severe neurotrophic keratitis, comparing it against a preservative-free artificial tear vehicle. The European study, NGF0212, randomly assigned 52 patients to treatment groups. After 8 weeks of treatment, approximately 72% of patients in the treatment group achieved complete corneal healing. The US-based study, NGF0214, randomly assigned 24 patients to treatment groups, with 65.2% of treated patients achieving complete corneal healing. Longer-term follow-up from the REPARO study revealed that approximately 82% of patients who achieved healing after the 8-week treatment course maintained stable healed status at one year.

Cenegermin was well tolerated in clinical trials. Ocular pain was the most common adverse reaction, reported by approximately 16% of patients, which may be associated with the restoration of sensation lost due to the disease. Other adverse events reported by more than 5% of patients included ocular swelling (inflammation), increased lacrimation (increased tear production), and ocular hyperemia.

Original Title: New Drug Urgently Needed in Clinical Practice! Cenegermin Eye Drops Approved in China for the Treatment of Neurotrophic Keratitis