Long-term data reinforce voretigene neparvovec-rzyl’s role in treating genetic retinal disorders

Voretigene neparvovec-rzyl (VN) gene therapy demonstrates sustained safety and efficacy in improving mobility and visual function for up to 9 years in patients with biallelic RPE65 mutation-associated retinal dystrophy, with no serious adverse events related to the therapy, according to a presentation at ASRS 2024.

This Phase 3 study compared outcomes between patients receiving VN at baseline (original intervention, OI) versus those receiving it after a 1-year delay (delayed intervention, DI).

The study included 29 patients, with 20 receiving VN at baseline and 9 undergoing delayed treatment. The primary efficacy measure was the Multi-luminance Mobility Test (MLMT), assessing improvements in mobility. Secondary endpoints included changes in full-field light sensitivity threshold (FST) and best-corrected visual acuity (BCVA).

Results demonstrated that VN therapy led to meaningful and sustained improvements in both mobility and visual function. At Year 5, OI patients showed a mean MLMT change score of 1.6, while DI patients had a score of 2.4. These improvements were maintained, with MLMT change scores of 1.5 at Year 8 and 2.2 at Year 9 for the combined OI and DI groups. Similarly, FST and BCVA measures showed continued benefits over the study period.

The safety profile of VN was consistent with previous findings. No serious adverse events related to VN were reported, although one DI patient experienced a serious adverse event of acute myeloid leukemia, deemed unlikely to be related to VN.

Reference
Russell S. Safety and Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 8 Years and 9 Years. Presented at: ASRS 42nd Annual Meeting: July 17–20, 2024.