voretigene neparvovec (Luxturna)

following reassessment through the ultra-orphan framework:

voretigene neparvovec (Luxturna®) is accepted for use within NHSScotland.

Indication under review: 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.

In a phase III open-label study of patients with vision loss due to inherited retinal dystrophy due to RPE65 mutations, functional vision was significantly improved from baseline to one year in the voretigene neparvovec group compared with the control group.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.