4.1 Therapeutic indications
Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Why sign up with Medthority?
Develop your knowledge with our disease and condition focused Learning Zones
Access content from credible sources, including expert-led commentary, videos, podcasts, and webinars as well as clinical trials, treatment information and guidelines
Personalised dashboard providing updates and recommendations for content within your areas of interest
Voretigene neparvovec (Luxturna®), a recombinant adeno-associated virus vector-based gene therapy, delivers a functioning copy of the human retinal pigment epithelium-specific 65 kDa (RPE65) gene into retinal cells of patients with reduced or absent levels of RPE65 protein, providing the potential to restore the visual cycle.
Purpose: To report the durability of voretigene neparvovec-rzyl (VN) adeno-associated viral vector-based gene therapy for RPE65 mutation-associated inherited retinal dystrophy (IRD), including results of a phase 1 follow-on study at year 4 and phase 3 study at year 2.
The drug SPC information (indications, contra-indications, interactions, etc), has been developed in collaboration with eMC (www.medicines.org.uk/emc/). Medthority offers the whole library of SPC documents from eMC.
Medthority will not be held liable for explicit or implicit errors, or missing data.
Drugs appearing in this section are approved by UK Medicines & Healthcare Products Regulatory Agency (MHRA), & the European Medicines Agency (EMA).