Study investigates safety, efficacy of Luxturna in real-world clinical practice

ByMichela Cimberle

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Initial findings from the real-world PERCEIVE study of voretigene neparvovec in patients with RPE65 mutation-associated inherited retinal dystrophy show safety and efficacy in line with those observed in pivotal trials.

“Despite limited availability of data at the time of this first analysis, the safety and tolerability of [voretigene neparvovec] observed in patients are consistent with its known safety profile,” M. Dominik Fischer, MD, said at the virtual Euretina congress.

Genes for ET weekender 11.2.2018 — *Source: Adobe Stock*

Inherited retinal dystrophy caused by biallelic RPE65 mutation is a rare genetic condition that can ultimately result in complete blindness. Luxturna (voretigene neparvovec, Spark Therapeutics/Novartis) is the first approved ocular gene therapy available for treating this disease in eyes with sufficient viable retinal cells.

“Current evidence is from trials, which include a limited number of patients, with scarce and no real-word data on its safety and effectiveness,” Fischer said.

PERCEIVE is an ongoing global registry-based post-authorization study designed to evaluate the real-world long-term safety profile of Luxturna via systematic collection of adverse event data, to follow pregnancy outcomes in patients receiving the treatment and to assess visual function over 5 years. The 6-month outcomes of the first 15 patients were presented at the meeting.

“Ocular adverse events were reported in four eyes of four patients and included dyschromatopsia, glare, and macular scar and foveal thinning, likely due to the subretinal administration procedure rather than to the drug,” Fischer said.

Substantial improvement in the full-field light sensitivity threshold test was observed in patients more than 6 months after receiving voretigene neparvovec, but no considerable changes were seen in mean foveal thickness and best corrected visual acuity.

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Source:

Fischer MD. Safety and effectiveness of voretigene neparvovec in patients with RPE65 mutation-associated inherited retinal dystrophy: initial findings from the ongoing, real-world, perspective, observational PERCEIVE study. Presented at: Euretina congress; Sept. 9-12, 2021 (virtual meeting).

Disclosures: Fischer reports receiving consulting fees from Adelphi Values, Advent France Biotechnology, Alphasight, Arctos Medical, Atheneum, Axiom Healthcare Strategies, Biogen, Cambridge Consultants, Decision Resources, Dialectica, Frontera Therapeutics, Janssen, Navigant, Novartis, Roche, Regenxbio, Sirion and STZ eyetrial.