LUXTURNA (voretigene neparvovec) - Inherited retinal dystrophy

The Committee deems that the clinical benefit of  LUXTURNA 5 x 1012 vector genomes/mL concentrate and solvent for solution for injection remains substantial in the MA indication.

Considering:

• the unmet medical need in an incapacitating disease that progresses to blindness in the absence of other curative treatments,
• the quality of the evidence of the efficacy of LUXTURNA (voretigene neparvovec) in randomised, multicenter phase 3 study 301 versus the absence of treatment, and despite the open-label nature of the study,
• the relevance of the primary endpoint assessing the visual function of patients using a Multi-Luminance Mobility Test (MLMT) and the clinical relevance of the effect observed after 1 year of follow-up compared to the absence of treatment,
• long-term follow-up data from 3 studies (extension of study 301 with usable data for up to 6 years of follow-up, the PASS PERCEIVE study based on data from the international registry of 198 patients with follow-up for 1-2 years or 1 additional year for French patients and the LIGHT observational study in 12 patients with follow-up for ≥ 1 year) demonstrating the maintenance of clinical responses in mobility tests, full-field light sensitivity threshold (FST) testing using white light and visual field,
• a safety profile that is unchanged overall since the product was first marketed,

despite:

• the absence of robust evidence of an effect of LUXTURNA (voretigene neparvovec) on quality of life,
• the development in some patients included in the clinical studies (frequency unknown according to the SmPC) of progressive chorioretinal atrophy, which had little impact on assessment of the endpoints but in which the long-term progression needs to be evaluated,

the Committee deems that the clinical added value of LUXTURNA 5 x 1012 vector genomes/mL concentrate and solvent for solution for injection remains important (CAV II) in the management 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.