Luxturna shows long-term maintenance of ambulatory vision, visual field
Luxturna maintained long-term improvements in ambulatory vision, light sensitivity and visual field in patients with biallelic RPE65 mutation-associated retinal diseases, according to a study.
The phase 3 study was designed to investigate the long-term efficacy and safety of Luxturna (voretigene neparvovec-rzyl, Spark Therapeutics) in two cohorts: an original intervention group that underwent a 9-year follow-up (20 patients) and a delayed intervention group that underwent an 8-year follow-up (nine patients), Stephen R. Russell, MD, of the University of Iowa, said at the American Society of Retina Specialists annual meeting.
The study’s primary endpoint was change in bilateral performance as assessed through a multi-luminance mobility test (MLMT), which measured patients’ functional vision at light levels ranging from 1 lux to 400 lux. To pass the test, participants were required to complete a course in less than 3 minutes with fewer than three errors.
After treatment with Luxturna, the MLMT test maintained ambulatory vision improvements through years 8 and 9. Improvements in full-field light sensitivity threshold (FST) and visual field as measured by Goldmann perimetry were also maintained through last follow-up.
“The FST remained greater than 1 log unit, near 2 log units, above preintervention,” Russell said. “But the visual field variability increases after 5 years, making it difficult to determine stability, although it remains above preintervention values.”
Mean best corrected visual acuity was stable through last follow-up for the combined cohorts with increased variability after year 5, which Russell noted as being due in part to missed appointments during the COVID-19 pandemic.
The most common adverse events included the development of late cataracts (28%) and increases in IOP (17%). Five patients in the clinical development program, which included phase 1 through phase 3 data, experienced depigmentation or pigmentary changes after subretinal injection that were attributed to natural disease progression.
“Adverse events were largely related to vitrectomy and subretinal injection procedures,” Russell said.
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Russell SR, et al. Safety and durability of voretigene neparvovec for biallelic RPE65-mediated inherited retinal disease: Phase 3 results at 8 years and 9 years. Presented at: American Society of Retina Specialists annual meeting; July 17-20, 2024; Stockholm.