Luxturna, a one-shot gene therapy product

Novartis Korea submits supplementary data to the HIRA

3월 약평위서 비급여 판정…RSA 조건서 이견

Attention is focusing on whether progress will be made in the discussion on insurance benefits for Luxturna, a one-shot retinal disease treatment.

 

As a result of the coverage, Novartis Korea recently submitted additional supplemental data for Inherited Retinal Dystrophy treatment Luxturna requested by the HIRA.

 

The drug submitted an application for reimbursement in September 2021, but there was no progress in the listing process, and it was presented to the Pharmaceutical Reimbursement Evaluation Committee in March but failed to set reimbursement standards.

 

It is known that during the evaluation process of Luxturna, there was a difference of opinion over the terms of the Risk Sharing Agreement (refund rate, etc.).

 

As Novartis submitted supplementary data and both the government and pharmaceutical companies are showing their will to be listed as Luxturna, it remains to be seen whether this re-challenge will be successful.

 

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.

 

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

 

Meanwhile, 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 for 1 year.

 

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