Three-year interim results of the PERCEIVE study


Voretigene neparvovec (Luxturna, Spark Therapeutics, Inc.) was shown to be safe and effective in real-world settings.

Image credit: AdobeStock/Curioso.Photography

(Image credit: AdobeStock/Curioso.Photography)

The 3-year interim results of the PERCEIVE Study, a large international trial, found voretigene neparvovec (Luxturna, Spark Therapeutics, Inc.) to be safe and effective in real-world settings, which agreed with its known safety profile in patients with RPE65-associated inherited retinal dystrophies. In addition, in most patients the visual function continued to improve, including in those with chorioretinal atrophy.

M. Dominik Fischer, MD, from the Center for Ophthalmology, University of Tubingen, Germany, and the Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK, reported the results for the study group at the Association for Research in Vision and Ophthalmology annual meeting in Seattle.

All of the study patients either had been treated with or were planning to be treated with voretigene neparvovec in at least 1 eye. The patients were followed for 5 years. The primary study objective was to collect information on both adverse events and adverse effects of special interest; the secondary objectives were the results of the assessment of visual function, Fischer recounted.

Interim results of voretigene neparvovec assessment

At the 3-year time point, 198 patients (mean age, 22.9; 53.5% male; mean follow-up, 1.4 years; maximal follow-up 3.4 years) had been treated with voretigene neparvovec.

Regarding ocular adverse effects, the authors reported that 123 patients (62.1%) had reported at least 1 adverse effect. Twenty-two patients had non-ocular adverse effects. A total of 109 patients (55.1%) reported ocular adverse effects of special interest that included chorioretinal atrophy in 49 patients (with retinal degeneration and injection site atrophy the most frequent), increased intraocular pressure in 32, intraocular inflammation and/or procedure-related infection in 28, cataract in 26, and foveal thinning in 12. Ocular serious adverse effects developed in 10 patients that included retinal foveal disorder and reduced visual acuity in 2 patients each.

The authors reported that, overall, the visual function improved. The mean changes in the full-field stimulus testing over baseline at years 1, 2, and 3 were as follows: it increased, respectively, to −18.41 decibels in 96 patients, to −13.37 decibels in 39 patients, and to −14.73 decibels in 10 patients. In addition, the mean best-corrected visual acuity observed out to year 3 did not decrease and the visual function improved in patients with chorioretinal atrophy.

The investigators concluded that the 3-year interim analysis showed the safety and effectiveness of voretigene neparvovec that agreed with its known safety profile. “Most patients had continued improvement in visual function, including those with chorioretinal atrophy. The etiology of the chorioretinal atrophy is unclear and may be multifactorial within the context of natural history, where retinal degeneration is a hallmark of disease,” they concluded

Fischer MD, Simonelli F, Audo IS, et al. Real-world safety and effectiveness of voretigene neparvovec: Year 3 interim results from the PERCEIVE study. Paper presented at: Association for Research in Vision and Ophthalmology annual meeting; May 5-9, 2024; Seattle, WA. Presentation number 3290
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