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. |