Novartis announces landmark EU approval for one-time gene therapy Luxturna to restore vision in people with rare inherited retinal disease
- Luxturna (voretigene neparvovec) is the first gene therapy to treat an inherited retinal disease, indicated for children and adults with vision loss caused by mutations in both copies of the RPE65 gene and sufficient viable retinal cells1
- Nearly 60% of patients have severe forms of the disease, with severe visual impairment occurring shortly after birth2
- As a one-time treatment, Luxturna restores vision and improves sight in children and adults with a sustained effect and favorable safety profile3,4
- Luxturna? (voretigene neparvovec) Novartis Pharmaceuticals. Approved EU SmPC. Available imminently at: https://www.ema.europa.eu/en/medicines
- Luxturna. BLA Clinical Review Memorandum. Yao-Yao Zhu. US Food and Drug administration. Available at: https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM592766.pdf. Last accessed on 1 November 2018
- Russell S et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65- mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. The Lancet 2017; 390:849-860.
- Russell S et al. Three-year update for the phase 3 voretigene neparvovec study in biallelic RPE65 mutation-associated inherited retinal disease. Investigative Opthalmology & Visual Science, 2018; 59
- Banken, R et al. Voretigene Neparvovec for Biallelic RPE65-Mediated Retinal Disease: Effectiveness and Value, Final Evidence Report. Institute for Clinical and Economic Review 2018. Available at https://icer-review.org/wp-content/uploads/2017/06/MWCEPAC_VORETIGENE_FINAL_EVIDENCE_REPORT_02142018.docx.pdf. Last accessed 1 November 2018.
- RetNet. Summaries of genes and loci causing retinal diseases. Available at: https://sph.uth.edu/retnet/sum-dis.htm. Last accessed November 2018
- Data on file. 2018
- Astuti GD et al. Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark. European Journal of Human Genetics 2016; 24: 1071?79.
- Morimura H et al. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis. Proceedings of the National Academy of Sciences of the USA. 1998; 95: 3088?93.