Topical Cenegermin for Neurotrophic Keratopathy
Ophthalmology, January 2020
Pflugfelder et al. evaluated the efficacy and safety of topical cenegermin in patients with neurotrophic keratopathy. They found that, when compared to vehicle, the drug was more effective at reducing lesion size and led to fewer events indicating disease progression. Moreover, cenegermin was well tolerated; most adverse effects were local, mild, and transient.
This double-masked, vehicle-controlled trial included 48 patients treated at 11 study sites in the United States. Participants were assigned randomly (1:1) to receive topical cenegermin 20 μg/mL or vehicle eye drops. Six drops were administered daily for eight weeks, and follow-up continued through 24 weeks. The primary end point was healing of the neurotrophic lesion (persistent epithelial defect or corneal ulcer) by week 8. Masked central readers measured neurotrophic lesions from clinical photographs and then assessed corneal healing status conventionally (<0.5 mm of fluorescein staining in greatest dimension of lesion area) as well as conservatively (0-mm lesion staining and no other residual staining).
Secondary outcomes included corneal healing at four weeks of treatment and overall changes in lesion size, disease progression events, visual acuity, and corneal sensitivity from baseline to week 8.
The conventional assessment showed significant differences at week 8, as 70% of the active-treatment arm and 29% of the vehicle arm had less than 0.5 mm of lesion staining (p = .006). With regard to the conservative assessment at week 8, 65% of the cenegermin group and 17% of those who received vehicle had 0 mm of lesion staining and no other staining residually (p < .001). Conservative evaluation also revealed significant between-group differences at week 4 (key secondary end point). Cenegermin produced significant reductions in lesion size and disease progression rates throughout treatment and was well tolerated. Most adverse events were mild, local, and resolved rapidly.
The authors concluded that cenegermin 0.002% ophthalmic solution represents a safe noninvasive option to treat neurotrophic keratopathy. They added that it “can become part of the treatment algorithm for this often difficult to manage disease with a high need for targeted and effective pharmacotherapies.” More research is warranted to define the pathologic processes modulated by cenegermin.
The original article can be found here.