Demonstration of the efficacy and safety of voretigene neparvovec-rzyl (in patients with RPE65-mediated Leber congenital amaurosis (LCA)1 represents a landmark—the first approved gene therapy in ophthalmology. The primary outcome measure (multiluminance mobility testing) was significantly better with voretigene neparvovec-rzyl compared with the untreated group receiving standard care at 1 year. The trial reported a “directional improvement” in visual acuity, but the differences between voretigene neparvovec-rzyl and the control group were not statistically significant. The trial also reported signs of improvement in other tests scaled to deficits in low-light and peripheral vision. Clearly, something positive was happening, and the researchers are to be praised for their ongoing seminal work.
Yannuzzi NA, Smiddy WE. Cost-effectiveness of Voretigene Neparvovec-rzyl Therapy. JAMA Ophthalmol. 2019;137(10):1123–1124. doi:https://doi.org/10.1001/jamaophthalmol.2019.2517
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