Purpose : To present 5-year post-injection results for the multi-luminance mobility test (MLMT) and full field light sensitivity threshold test (FST) endpoints in the original intervention and delayed intervention groups of the phase 3 voretigene neparvovec-rzyl (VN) study in biallelic RPE65 mutation–associated inherited retinal disease.
Methods : Patients were randomized to either original intervention (OI:bilateral subretinal VN at baseline; n=20) or delayed intervention (DI:VN after 1 year; n=9). The primary endpoint was Multi-Luminance Mobility Test (MLMT) at 7 standard light levels as measured by a change score. The first secondary endpoint was full-field light sensitivity threshold (FST). The five-year timepoint was analyzed for OI and DI groups individually and combined OI group plus DI group.
Results : For OI patients at Year 5 (n=18) and DI patients at Year 5 (n=7), the MLMT mean (SD) bilateral light level change score was 1.6 levels (1.2) and 2.4 levels (1.6), respectively compared to baseline. The combined (OI plus the DI groups) mean bilateral light level change score was 1.8 (1.3) (N=25 ). Mean change in white light FST in log10 (cd.s/m2) was −2.02 (1.45) log10 at Year 5 for OI patients (n=17) and −2.57 (1.21) log10 at Year 5 for DI patients (n=7). The mean change in white light FST in log10 (cd.s/m2) was -2.18 (1.38) for the combined OI and DI group (N=24).
Conclusions : Visual improvements are maintained for at least 5 years after VN administration for both the OI and DI patients