June 2021
Volume 62, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2021
Five-Year Post-Injection Results of the Phase 3 Trial of Voretigene Neparvovec-rzyl in Biallelic RPE65 Mutation-Associated Inherited Retinal Disease
Author Affiliations & Notes
  • Jean Bennett
    Ophthalmology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
    Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States
  • Stephen R Russell
    University of Iowa, Iowa City, Iowa, United States
  • Katherine A High
    Spark Therapeutics, Inc (formerly), Philadelphia, Pennsylvania, United States
  • Arlene V Drack
    University of Iowa, Iowa City, Iowa, United States
  • Zi-Fan Yu
    Statistics Collaborative, Inc., Washington, District of Columbia, United States
  • Daniel C Chung
    Spark Therapeutics, Inc., Philadelphia, Pennsylvania, United States
  • Kathy Reape
    Spark Therapeutics, Inc (formerly), Philadelphia, Pennsylvania, United States
  • Albert M Maguire
    Ophthalmology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
    Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States
  • Footnotes
    Commercial Relationships   Jean Bennett, Spark Therapeutics, Inc. (F); Stephen Russell, Novartis (C), Spark Therapeutics, Inc. (F); Katherine High, Spark Therapeutics, Inc. (C), Spark Therapeutics, Inc. (former relationship) (E); Arlene Drack, ProQr (F), ProQr (advisory board) (C), Retophin (F), Spark Therapeutics, Inc. (F); Zi-Fan Yu, Spark Therapeutics, Inc. (through employer) (C); Daniel Chung, Spark Therapeutics, Inc. (E); Kathy Reape, Spark Therapeutics, Inc. (C), Spark Therapeutics, Inc. (former relationship) (E); Albert Maguire, Spark Therapeutics, Inc. (F)
  • Footnotes
    Support  This study was sponsored by Spark Therapeutics, Inc.
Investigative Ophthalmology & Visual Science June 2021, Vol.62, 3540. doi:
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      Jean Bennett, Stephen R Russell, Katherine A High, Arlene V Drack, Zi-Fan Yu, Daniel C Chung, Kathy Reape, Albert M Maguire; Five-Year Post-Injection Results of the Phase 3 Trial of Voretigene Neparvovec-rzyl in Biallelic RPE65 Mutation-Associated Inherited Retinal Disease. Invest. Ophthalmol. Vis. Sci. 2021;62(8):3540.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

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.

This is a 2021 ARVO Annual Meeting abstract.

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