The FDA announced on Tuesday the approval of Luxturna to treat children and adult patients with inherited retinal dystrophy, specifically biallelic RPE65 mutation-associated retinal dystrophy. This marks another milestone in gene therapy and is the first time the FDA has approved a gene therapy for an inherited condition and reinforces the potential of gene therapy in treating a wide-range of difficult diseases.
From 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.
The accompanying vision loss often occurs during childhood and progresses to complete blindness. The retinal dystrophy is caused by defects in a gene involved in producing a protein critical to normal vision. Luxturna uses reengineered viruses to insert a copy of the gene into retinal cells in order to produce the necessary protein and restore vision. The breakthrough treatment was unanimously approved by the overseeing advisory committee.
While Luxturna is going to provide great relief in a disease that has few options, the cost to patients is a point of concern. The manufacturer, Spark Therapeutics, hasn't revealed the list price of the treatment, and won't do so until January, but many are expecting a hefty price tag to come with this novel treatment with estimates ranging up to $1 million per patient. How they price the treatment, as well as what stances managed care organizations and treatment centers adopt toward the cost of this treatment will undoubtedly be watched very carefully.
The treatment is called Luxturna, a genetically modified virus that ferries a healthy gene into the eyes of patients born with retinal dystrophy, a rare condition that destroys cells in the retina needed for healthy vision. In tests on patients, the treatment often produced dramatic results, restoring the ability of patients to see things they could never see before, such as the stars, the moon, fireworks and their parents' faces. The treatment also enabled patients to do many things that had been impossible, such as read, play sports, ride bicycles and go outside at night by themselves.