Application Detail
Description of Medical Service
Vortigene neparvovec (VN) is a recombinant adeno-associated viral serotype 2 (AAV2) gene therapy which must be delivered in close proximity to the retinal pigment epithelium (RPE) target cells. To access the retina, the vitreous gel that fills the eye is completely removed through a vitrectomy. VN is then injected into the space between the retina and the RPE, as a “subretinal” injection.Description of Medical Condition
Biallelic RPE-65-mediated inherited retinal dystrophies (IRD), the most severe form of IRD, are a major cause of early-onset blindness. They occur when when there are mutations in both alleles of the RPE65 gene in RPE cells.Reason for Application
Health technology assessmentMedical Service Type
TherapeuticPrevious Application Number/s
Not applicableAssociated Documentation
Application Form
-Consultation Survey
Consultation Survey (PDF 561 KB)Consultation Survey (Word 70 KB)
PICO Confirmation
-Assessment Report
-Public Summary Document
Public Summary Document (PDF 1656 KB)Public Summary Document (Word 529 KB)
Meetings for this Application
PASC
-ESC
12 June 2020MSAC
28-29 July 202026-27 November 2020