Spark Therapeutics (ONCE) Gets Approval for First Gene Therapy for a Genetic Disease

Edward Kim  |

The FDA approved a new gene therapy to treat an inherited form of vision loss that may result in blindness. The drug, by Spark Therapeutics (Nasdaq: ONCE), is called Luxturna (voretigene neparvovec-rzyl), and is the first directly administered gene therapy approved in the US that targets a disease caused by mutations in a specific gene.

Luxturna was approved to treat adults and children with treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. In addition to being the first approved gene therapy for a genetic disease, it is the first pharmacologic treatment for an inherited retinal disease and the first adeno-associated virus (AAV) vector gene therapy approved in the US.

The approval was welcome news to Spark Therapeutics and its shareholders, who were shaken last week when the company's Phase I/II data for its hemophilia A treatment was below expectations. The stock dropped 35% on that news and has held this level since then.

According to the FDA, "hereditary retinal dystrophies are a broad group of genetic retinal disorders that are associated with progressive visual dysfunction and are caused by mutations in any one of more than 220 different genes. Biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the US... Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision."

Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells, spurring the production of the normal protein that converts light to an electrical signal in the retina to restore a patient’s vision loss. Luxturna uses a naturally occurring AAV, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene.

This one-time gene therapy for an inherited disease represents a first-of-its-kind breakthrough that may lay the groundwork for the development of gene therapies for other conditions that are not adequately addressed today.

- Jeffrey D. Marrazzo, CEO, Spark Therapeutics

Finally, Spark Therapeutics received a Rare Pediatric Disease Priority Review Voucher by the FDA, which can be redeemed by a sponsor at a later date to receive Priority Review of a subsequent marketing application for a different product. This is the 13th rare pediatric disease priority review voucher issued by the FDA since the program began.

Spark Therapeutics Pipeline



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