• Topical Cenegermin for Neurotrophic Keratopathy

    Written By: Lynda Seminara
    Selected By: Stephen D. McLeod, MD

    Journal Highlights

    Ophthalmology, January 2020

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    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.