CADTH Canadian Drug Expert Committee recommendation: Voretigene neparvovec (Luxturna -- Novartis Pharmaceuticals Canada Inc.) indication : vision loss, inherited retinal dystrophy

Voretigene neparvovec has a Health Canada indication for the treatment of adult and pediatric patients with vision loss due to IRD caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. Voretigene neparvovec is a vector-based gene therapy designed to deliver a no...

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Bibliographic Details
Corporate Authors: CADTH Canadian Drug Expert Committee, Canadian Agency for Drugs and Technologies in Health
Format: eBook
Language:English
Published: Ottawa (ON) Canadian Agency for Drugs and Technologies in Health November 2020, 2020
Edition:Version: 1.0
Series:CADTH common drug review
Online Access:
Collection: National Center for Biotechnology Information - Collection details see MPG.ReNa
Description
Summary:Voretigene neparvovec has a Health Canada indication for the treatment of adult and pediatric patients with vision loss due to IRD caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. Voretigene neparvovec is a vector-based gene therapy designed to deliver a normal copy of the gene encoding the human RPE65 to cells of the retina in persons with reduced or absent levels of biologically active RPE65. It is available as vector genomes/mL concentrate for solution for subretinal injection and the Health Canada-approved dose is 1.5 x 1011 vector genomes (vg) for each eye
Item Description:"Final."
Physical Description:1 PDF file (10 pages)