TGA approved Novartis Luxturna in Australia
Updated: Aug 21, 2020
Australian Therapeutic Goods Administration has approved Luxturna (voretigene neparvovec) for treating patients with inherited retinal dystrophy because of biallelic RPE65 mutations.
Luxturna is gene therapy; the drug acts by delivering a copy of the gene that codes the retinal pigment epithelial 65 kDa protein in patients who lacks biologically active RPE65. Patients with inherited retinal dystrophy have visual impairment because of biallelic RPE65 mutation, which leads to reduced or absence of RPE65 isomerohydrolase activity. This impacts the visual cycle.
The efficacy of Luxturna was evaluated in 31 patients. Of the 31 patients, Luxturna was administered in 21 patients. Ten subjects were included as a control group, among whom nine patients were randomized and treated with Luxturna after one year of observation. The efficacy was measured using the multi-luminance mobility testing (MLMT) score. Eleven of twenty-one patients who were on Luxturna had an MLMT score change of two or more, whereas only one patient in the control group had a difference in the MLMT score.
The drug is approved in the black triangle scheme. Luxturna received orphan designation in 2019.
The product has already approved in other major markets, including the United States, Europe.
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