Brand Name



Recombinant human nerve growth factor

FDA Indication

Neurotrophic keratitis†,$


Cenegermin is recombinant human nerve growth factor produced in E. coli bacteria.10 It is identical to the endogenous human protein, and FDA approved it as a breakthrough therapy for the treatment of neurotrophic keratitis (NK). NK is a degenerative eye disease caused by impaired trigeminal innervation. It is characterized by reduced or absent corneal sensation, reduction in neuromodulators, loss of protective reflexes, epithelial breakdown, and impaired healing.73 Patients with NK may develop recurrent or persistent corneal ulceration, “melting,” and perforation.73,74 Corneal perforation may lead to scaring and permanent vision loss.74 The most common triggers for the development of NK are chemical burns, chronic use of topical ophthalmic medications (e.g., anesthetics, beta blockers, sulfonamides, and NSAIDs), diabetes, herpesvirus infections, multiple sclerosis, physical injuries, and tumors.73,74

The traditional management for NK has been palliative care with artificial tears for lubrication and prophylactic antibiotic eye drops. Unproven therapy with specialty compounded serum-derived eye drops has been advocated (NCT03085290).75-77 Nonpharmacological treatments include the use of bandage-type contact lenses and, in the most serious cases, surgery to create a conjunctival flap or protective closure of the eyelid through either a manual (i.e., tarsorrhaphy) or a pharmacologic-based procedure (i.e., injection of botulinum A toxin).74,77

Nerve growth factor binds to neurotrophic tropomyosin receptor kinase A (TrkA) and nerve growth factor receptor p75 to stimulate signaling pathways involved in the normal differentiation, development, repair, and survival of sympathetic and sensory neurons.10,78 Cenegermin is formulated as an ophthalmic solution for administration at a dose of one drop in the affected eye every two hours, for a total of six times daily. The most common adverse reactions reported with cenegermin therapy are eye pain, corneal deposits, foreign body sensation, ocular hyperemia, ocular inflammation, and tearing.

Through topical application to the eye, cenegermin acts on receptors in the anterior segments (i.e., iris, ciliary body, lens, cornea, and conjunctiva) to increase tear film production and promote corneal epithelial healing.10,74 In premarket studies of patients with NK and persistent corneal epithelial defects or ulcers, 70% of subjects treated with cenegermin achieved complete corneal healing within eight weeks versus 28% of patients treated with control drops lacking cenegermin. The average wholesale price for a 7-day course of therapy is $14,160.00.79

Further Reading in Goodman & Gilman’s