Luxturna, a one-shot retinal disease treatment

Apply immediately after failing to pass the committee last month

Reduce the risk of blindness

Luxturna, a one-shot retinal disease treatment, is once again aiming to enter insurance coverage.

 

As a result of the coverage, Novartis Korea recently resubmitted a reimbursement application for Luxturna, a treatment for Inherited Retinal Dystrophy.

 

This is a quick resumption of the process after the HIRA's non-reimbursed decision last month.

 

As the company's will to be listed on the salary is firm, it remains to be seen whether this re-challenge will be successful.

 

This drug submitted an application for reimbursement in September 2021, but there was no progress in the listing process so far, and it was first introduced this year.

 

Although it is expensive one-shot gene therapy, it seemed difficult to register because the disease is not directly related to life.

 

Luxturna restores the function of the defective or defective RPE65 gene, one of the causes of IRD, by replacing it with a normal gene with just one administration.

 

This means that the fundamental treatment of the disease is possible.

 

Therefore, the key is how much Luxturna can achieve the value of preventing blindness.

 

This drug was designated by the US FDA as Breakthrough Therapy in 2014, Orphan Drug in 2016, and Priority Review in 2017, and obtained expedited approval in 2017.

 

IRD is a rare and intractable disease in which vision loss occurs due to mutations in the gene responsible for the structure and function of retinal photoreceptors.

 

It includes more than 20 different eye diseases, and there are about 300 causative genes.

 

IRD caused by RPE65 gene mutation causes an abnormality in the visual cycle in the retina, which converts visual information entering the eye into nerve signals and transmits them to the brain.

 

RPE65 gene mutation reduces the RPE65 protein, which is essential for visual circuitry, and destroys retinal cells, gradually narrowing the field of vision and eventually leading to blindness.

 

Luxturna proved its effectiveness through a phase 3 clinical trial targeting patients with hereditary retinal diseases in which a biallelic mutation in the RPE65 gene was confirmed.

 

As a clinical result, the functional vision of patients treated with Luxturna improved statistically significantly compared to the control group who did not receive treatment at 1 year of treatment.

 

As a result of evaluating the average score of the Multi-Luminance Mobility Test (MLMT), which evaluates the ability to pass through an obstacle course of various heights in various levels of illumination by recreating a daily walking environment, as the primary evaluation variable at 1 year of treatment, Luxturna treatment group The score change of was 1.8 points, which was 1.6 points higher than the control group's score change of 0.2 points.