Oct 3, 2022

Will experience support use of first-ever retinal gene therapy?

by: Maximilian J. Gerhardt, MD

Recent reports of retinal atrophy have raised concerns on potential long-term safety.

Voretigene neparvovec (Luxturna, Spark Therapeutics) is the first causal treatment for biallelic RPE65 mutation–associated retinal disease, which regularly progresses to legal blindness. The one-time gene therapy aims to deliver the correct coding sequence of the human RPE65 gene to the retinal pigment epithelium and is performed via subretinal injection following vitrectomy.

The therapy was approved by both the European Medicines Agency (in 2018) and US Food and Drug Administration (in 2017) after data from the supporting pivotal phase 3 study revealed statistically significant functional vision improvement in patients in terms of increased light sensitivity. The findings also showed improved ability to navigate a mobility course at different levels of environmental illumination.

New gene therapies such as these raise the hope of treating a previously incurable disease with a favorable adverse effect profile. However, as with any new therapeutic product, there are limited real-world data, so it is natural that uncertainties regarding the durability and benefit-risk ratio exist. Recent reports of retinal atrophy development in the postoperative course of the disease have led to concerns that voretigene neparvovec could lead to potentially devastating consequences in the long-term…..

Read more: https://www.ophthalmologytimes.com/view/will-experience-support-use-of-first-ever-retinal-gene-therapy-

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