Voretigene neparvovec (Luxturna®) 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.
|NCPE Assessment Process||Complete|
|Rapid review commissioned||24/09/2019|
|Rapid review completed||23/10/2019|
|Rapid Review outcome||A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of voretigene neparvovec compared with the current standard of care.|