Applications

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

Find out about the service or technology in this application and the medical condition it addresses. You can also view the application documents, the deadlines for providing consultation input and the outcome of the application when the MSAC process is complete.

  • Status Complete
  • Type New application
  • Pre-PASC consultation -
  • Pre-MSAC consultation -
  • Outcome Supported

Application details

Reason for application

Health technology assessment.

Service or technology in this application

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.

Type: Therapeutic

Medical condition this application addresses

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.

Application documents

Consultation survey

Public summary document

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Meetings to consider this application

  • PASC meeting: -
  • ESC meeting: 12 June 2020
  • MSAC meeting: 
    • 28 to 29 July 2020
    • 26 to 27 November 2020