trending Market Intelligence /marketintelligence/en/news-insights/trending/XjPZP60IVBarWALpjVNA1A2 content esgSubNav
In This List

Spark's Luxturna wins FDA approval to treat rare form of inherited vision loss

Video

S&P Capital IQ Pro | Unrivaled Sector Coverage

Video

S&P Capital IQ Pro | Powering Your Edge

Blog

Enterprises are missing out on 24B by not optimizing cloud spending not going multicloud

Blog

Investment Research Analysts Providing Greater Coverage on Inflation


Spark's Luxturna wins FDA approval to treat rare form of inherited vision loss

Spark Therapeutics Inc.'s Luxturna drug secured U.S. Food and Drug Administration approval to treat patients with an inherited form of vision loss that may result in blindness.

The treatment will be used for children and adults with biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.

Luxturna, also known as voretigene neparvovec, delivers a normal copy of the RPE65 gene directly to retinal cells, which then produce normal proteins responsible for converting light into electrical signals in the retina to restore vision loss.

Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

Luxturna was awarded orphan designation by the U.S. FDA in January and later the regulator granted a priority review for the company's biologics license application.

Also, the drug awaits approval in the EU, where Spark submitted an application to the European Medicines Agency earlier in July.

Spark plans to disclose the price for the gene therapy in early January 2018, Reuters reported, citing an email from a company spokeswoman.