1623 - Voretigene neparvovec (Luxturna) for the treatment of biallelic RPE-65-mediated Inherited Retinal Dystrophies

Page last updated: 20 October 2020

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 assessment

Medical Service Type

Therapeutic

Previous Application Number/s

Not applicable

Associated Documentation

Application Form

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Consultation Survey

Consultation Survey (PDF 561 KB)
Consultation Survey (Word 70 KB)

PICO Confirmation

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Assessment Report

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Public Summary Document

Public Summary Document (PDF 960 KB)
Public Summary Document (Word 511 KB)

Meetings for this Application

PASC

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ESC

12 June 2020

MSAC

28-29 July 2020