- Luxturna is the first gene therapy treatment for an inherited genetic mutation
- The treatment is set to be available in the first part of 2018
(CNN)The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye disease.
Voretigene neparvovec, which will be sold as Luxturna, is made by Philadelphia-based Spark Therapeutics Inc.
The one-time treatment is approved for children and adults with retinal dystrophy due to a mutation of the RPE65 gene, which causes severe visual impairment beginning in infancy. As it progresses, patients experience gradual loss of peripheral and central vision, which can eventually lead to blindness.
There are more than 200 genes that can cause retinal dystrophy. Patients can be tested to determine whether the cause is indeed a mutation of the RPE65 gene and they are thus a potential candidate for this new treatment.