Regulatory Decision Summary for Oxervate

Authorization of Oxervate (cenegermin) eye drops for the treatment of moderate and severe neurotrophic keratitis (NK) was based on 2 Phase 1/2 vehicle-controlled, double-masked, randomized controlled trials in which 108 patients with moderate to severe NK received a treatment course of 0.002% Oxervate and 80 patients received vehicle eye drops. The treatment cycle consisted of one eye drop, 6 times per day in 2-hour intervals, for a period of 8 weeks. The primary efficacy endpoint(s) were complete healing as defined as either a persistent epithelial defect or corneal ulcer lesion <0.5 mm or as the lack of a detectable lesion upon assessment with fluorescein stain. There were significantly more stage 2 and 3 NK patients treated with Oxervate that achieved complete healing by both definitions as compared to patients treated with vehicle.

The most common adverse reactions (ADRs) were ocular in nature, and the most common adverse effect was eye pain/discomfort.

The recommended dose of 0.002% (20 mcg/mL) Oxervate is one eye drop in the affected eye(s), 6 times per day in 2 hour intervals, for a duration of 8 weeks.

Health Canada granted this submission priority review because it is intended to treat a rare disease for which no adequate medicinal treatment exists in Canada.

Overall, the benefits of treatment with Oxervate in patients with moderate or severe neurotrophic keratitis outweigh the risks. Appropriate labelling and risk management are considered adequate to address the risks and unknowns for this drug.