Currently, Oxervate is the only FDA-approved pharmaceutical treatment for NK. Oxervate is marketed by Dompe; the generic name is cenegermin. It is a recombinant human nerve growth factor formulated as an eye drop for use 6 times daily for 8 weeks. The clinical trial results that led to FDA approval can be found here:
Pflugfelder SC, Massaro-Giordano M, Perez VL, Hamrah P, Deng SX, Espandar L, Foster CS, Affeldt J, Seedor JA, Afshari NA, Chao W, Allegretti M, Mantelli F, Dana R. Topical Recombinant Human Nerve Growth Factor (Cenegermin) for Neurotrophic
Keratopathy: A Multicenter Randomized Vehicle-Controlled Pivotal Trial. Ophthalmology. 2020 Jan;127(1):14-26. doi: 10.1016/j.ophtha.2019.08.020.
Other eye drop treatments for NK are being investigated in clinical trials:
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Brim Biotech is testing BRM424, a regenerative agent (NCT05927428)
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Claris is testing CSB-001, a recombinant human hepatic growth factor (NCT04909450)
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Recordati Rare Diseases is testing REC 0/0559, also known as MT-8 or REC-0559, a human nerve growth factor mimetic (NCT04276558)
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RegeneRx is testing RGN-259, a thymosin beta4-based eye drop. The use of this eye drop for treating dry eyes is described in this article: Sosne G, Dunn SP, Kim C.Cornea. Thymosin β4 significantly improves signs and symptoms of severe dry eye in a phase 2 randomized trial. 2015 May;34(5):491-6. doi: 10.1097/ICO.0000000000000379.PMID: 25826322 Clinical Trial.
A number of centers are investigating eye drops formulated with insulin. For example:
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Krolo I, Behaegel J, Termote K, de Bruyn B, De Schepper M, Oellerich S, Ní Dhubhghaill S. The role of topical insulin in ocular surface restoration: A review. Surv Ophthalmol. 2024 Sep-Oct;69(5):805-817. doi: 10.1016/j.survophthal.2024.04.003.
A number of centers are investigating plasma rich in growth factors (PRGF), a blood product with concentrated levels of a person’s own growth factors that can be formulated into eye drops (i.e. NCT02707120).
For severe NK, a complex surgical technique known as corneal neurotization can take a sensory nerve from another part of the body to restore corneal sensation and promote corneal healing.
A detailed review of new therapies can be found here: Vera-Duarte GR, Jimenez-Collado D, Kahuam-López N, Ramirez-Miranda A, Graue-Hernandez EO, Navas A, Rosenblatt MI. Neurotrophic keratopathy: General features and new therapies.