Will Luxturna will be deliberated by DREC for reimb?

Attention is focused on whether the one-shot treatment Luxturna will be deliberated at the Drug Reimbursement Evaluation Committee meeting in March

Luxturna's reimbursement remains pending without much progress since the company applied for reimbursement in 2021

Can replace a defective gene with a single shot...reduces the risk of blindness

Industry attention is focused on whether progress will be made in discussions for the reimbursement of the one-shot retinal dystrophy treatment Luxturna.

 

It was found that it is highly likely that 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 next month (March).

 

Although the company applied for reimbursement of the drug in September 2021, no progress had been made on its listing since then.

 

Being a high-priced one-shot treatment for a non-life-threatening condition, these factors have acted as a barrier to the reimbursement listing until now.

 

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.

 

Therefore, the passage depends on how well the company conveys the value of Luxturna in preventing blindness to the authorities.

 

In the U.S., the drug was 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 intractable 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, there are currently over 300 genes that have been identified as being associated with IRD.

 

IRD, which is caused by the mutation in the 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 demonstrated through 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.