Real-world safety and effectiveness of voretigene neparvovec: Year 3 interim results from the PERCEIVE study

Abstract

Purpose : To present the interim Year 3 results of PERCEIVE study evaluating the long-term safety and effectiveness of Voretigene neparvovec (VN) in patients with RPE65-associated inherited retinal dystrophies with sufficient viable retinal cells in a real-world setting.

Methods : Patients who have received or have been planning to receive VN in at least 1 eye were enrolled. VN was administered according to local prescribing information and patients are followed up for 5 years. The primary objective was to collect information on adverse events (AEs) and AEs of special interest (AESIs). The secondary objectives are to assess pregnancy outcomes and visual function.

Results : At 3-year cut-off, 198 patients had received VN. The mean age (standard deviation [SD]) of patients was 22.9 (14.2) years; 106 (53.5%) were males. The mean (SD) length of follow-up was 1.4 (0.8) years (maximum: 3.4 years). At least 1 ocular AE was reported in 123 patients (62.1%). Ocular AESIs occurred in 109 patients (55.1%), including chorioretinal atrophy (n=49), increased intraocular pressure (n=32), intraocular inflammation and/or infection related to procedure (n=28), cataract (n=26), and foveal thinning (n=12). Ocular serious AEs (SAEs) were reported in 10 patients, including retinal fovea disorder (n=2) and reduced visual acuity (n=2). Among chorioretinal atrophy AESI, retinal degeneration (n=39) and injection site atrophy (n=15) were the most frequent. Twenty-two patients had non-ocular AEs. Overall, visual function was improved: mean (SD) change in FST at Year 1, 2 and 3 to −18.41 (14.66; n=96), −13.37 (19.56; n=39) and −14.73 (19.04; n=10) decibels, respectively, from baseline and no reduction in mean best-corrected visual acuity observed up to Year 3. Improvement in visual function was also observed for eyes of patients with chorioretinal atrophy.

Conclusions : Interim analysis of the PERCEIVE study at Year 3 demonstrates the safety and effectiveness of VN in line with the known safety profile of VN. A majority of patients showed continued improvement in visual function, including patients with chorioretinal atrophy. The etiology of the observed chorioretinal atrophy (AESI) remains unclear and may be multi-factorial within the context of natural history, where retinal degeneration is a hallmark of disease.

This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.