About My Health Careers Internship MedBlogs Contact us
Medindia LOGIN REGISTER
Advertisement

Gene Therapy For Blindness Costs 850,000 For Both Eyes

by Julia Samuel on January 8, 2018 at 12:56 PM
Font : A-A+

Gene Therapy For Blindness Costs 850,000 For Both Eyes

Spark Therapeutics, the manufacturer of the drug Luxturna which is said to treat retinal blindness comes with a costly price tag.

The drug will retail for $425,000 (Rs 2,69,89,625) per eye which is close to a million for both eyes and has raised a debate in the US for the supposedly innovative treatment. The gene therapy repairs a defective gene that causes retinal blindness.

Advertisement


It is the first gene therapy to be approved for use in the US and was given the go-ahead by the Federal Drug Administration in mid-December, 2017. "We believe that price reflects the type of life-altering value we're seeing with Luxturna in clinical trials and will allow us to build on revolutionary science," Spark Chief Executive Jeff Marrazzo

How Does The Drug Work?

The drug directly delivers a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore the patient's vision loss.
Advertisement

Bi-allelic RPE65 mutation-associated retinal dystrophy affects around 1,000 to 2,000 patients in the U.S. The RPE65 gene encodes for a protein that is essential for normal vision. Mutations in this RPE65 gene lead to reduced or absent levels of RPE65 protein activity, affecting the visual cycle and resulting in impaired vision.

Children with the gene mutation often are diagnosed at an early age with disorders such as Leber congenital amaurosis (LCA) or retinitis pigmentosa. Affected individuals experience progressive loss of vision over time. This loss of vision, seen typically during childhood or adolescence, ultimately progresses to complete blindness.

Results of The Trial

Within weeks of receiving the injections, most of them had improved vision; in fact, half of them had improvements to the degree they were no longer considered legally blind.

Results of the phase III trial showed improved ability to navigate in low light, increased sensitivity to light and better side vision. Many of the volunteers are now able to recognize faces, read chalkboard, do grocery shopping, take driving tests, and have more job opportunities, all of which seemed an impossible dream earlier.

Anticipating criticism, Spark promised to reimburse patients if the treatment proves not to be effective, and said it expects US health authorities to authorize the payment in stages. There are currently about 1,000 cases of people who suffer from hereditary degeneration of the retina, and 10 to 20 new cases are expected to be added each year. The FDA stated that the gene therapy should be given only to patients who have viable retinal cells as determined by the physicians.

Source: Medindia
Advertisement

Advertisement
News A-Z
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Advertisement
News Category
What's New on Medindia
International Day of Persons with Disabilities 2021 - Fighting for Rights in the Post-COVID Era
Effect of Blood Group Type on COVID-19 Risk and Severity
Woman with Rare Spinal Cord Defect from Birth Sues Doctor
View all

Medindia Newsletters Subscribe to our Free Newsletters!
Terms & Conditions and Privacy Policy.

More News on:
DNA Finger Printing Nervous Tic Reiki and Pranic Healing Genetic Testing of Diseases Pancreas Blindness Facts Best Disease Weaver Syndrome Usher Syndrome Trachoma 

Recommended Reading
Retinitis Pigmentosa
Retinitis pigmentosa is an inherited degenerative disorder which affects retina's ability to ......
Night Blindness
Night blindness is a common result of vitamin A deficiency, which is preventable and reversible....
Anophthalmia
Anophthalmia is a genetic condition where a baby is born without one or both eyes. Treatment is ......
Best Disease
People with Best disease may be completely normal or may suffer from blindness in due course....
DNA Finger Printing
DNA fingerprinting is a technique which helps forensic scientists and legal experts solve crimes, id...
Genetic Testing of Diseases
Genetic testing helps to confirm a genetic condition in an individual and involves q complex laborat...
Nervous Tic
Trigeminal Neuralgia or tic douloureux is one of the most painful nervous system disorders that affe...
Trachoma
Trachoma is a bacterial eye infection. It is the main cause of irreversible blindness due to infecti...
Usher Syndrome
Usher syndrome is a rare genetic disease which is the leading cause of deaf-blindness in humans....
Weaver Syndrome
Weaver syndrome is a genetic disorder in which children show accelerated bone growth, advanced bone ...

Disclaimer - All information and content on this site are for information and educational purposes only. The information should not be used for either diagnosis or treatment or both for any health related problem or disease. Always seek the advice of a qualified physician for medical diagnosis and treatment. Full Disclaimer

© All Rights Reserved 1997 - 2021

This site uses cookies to deliver our services. By using our site, you acknowledge that you have read and understand our Cookie Policy, Privacy Policy, and our Terms of Use