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Attention focused on whether Luxturna will be reimb in Korea
by Eo, Yun-Ho Nov 22, 2022 06:04am
Discussions on expanding coverage of the one-shot gene therapy ‘Luxturna’ is not making much progress. Therefore, the industry’s eyes are on whether Novartis Korea’s Inherited Retinal Dystrophy (IRD) treatment Luxturna (voretigene neparvovec) will be presented as an agenda for deliberation by the Health Insurance Review and Assessment Service’s Drug Reimbursement Evaluation Committee within this year. Novartis Korea applied for reimbursement of the drug in September last year, but no news has been heard on its passage of the Drug Reimbursement Standard Subcommittee until now. However, based on the speed of progress, the drug can still be listed in the first half of next year at the earliest. By replacing the defective or defective RPE65 gene - one of the causes of IRD - with a normal gene, Luxturna restores the visual function of an IRD patient with a single administration. In other words, the drug provides a fundamental cure for IRD. After being granted a Breakthrough Therapy Designation by the FDA in 2014, the drug was approved as an orphan drug in 2016, then granted priority review and a fast-track designation in 2017. IRD is a rare disease in which vision loss occurs due to mutations in genes responsible for the structure and function of retinal photoreceptors. In addition to over 20 ophthalmologic diseases, IRD leads to vision loss and may cause complete blindness in certain patients. There are currently over 300 genes that have been identified as being associated with IRD. IRD, which is caused by the mutation in RPE65 gene, causes abnormalities in the visual cycle of the retina that converts visual information into a neural signal and delivers it to the brain. The mutation in the RPE65 gene reduces the RPE65 protein essential to the visual cycle and destroys the retinal cell, gradually narrowing the field of vision to eventually result in blindness. Meanwhile, the efficacy of Luxturna was established in a Phase III trial that was conducted on IRD patients with confirmed biallelic RPE65 mutations. Study results showed that the group of patients that received Luxturna demonstrated statistically significant improvements in their functional vision compared to the control group at one year of treatment. Using the mean score of the multi-luminance mobility test (MLMT), which evaluates the ability to complete the obstacle course at low light levels by recreating the daily walking environment, as the primary endpoint at one year of treatment, the MLMT score change in the Luxturna treatment group was 1.8 points, which was 1.6 points higher than the 0.2 points in the control group.