June 2018

FDA Approves First Gene Therapy to Treat a Rare Form of Inherited Vision Loss

Luxturna is the first directly administered gene therapy approved in the United States that targets a disease caused by mutations in the RPE65 gene (RPE is retinal pigment epithelium).1 Luxturna is used to treat a rare, inherited form of vision loss caused by these mutations. A normal functioning RPE65 gene makes a protein that is essential for normal vision. Individuals with this inherited form of vision loss have defects (i.e., mutations) in both copies of the RPE65 gene which result in an absence or reduced level of the protein needed for normal vision.

Luxturna is expected to be available for administration in select treatment centers late in the first quarter of 2018.2 Luxturna will be covered under medical benefit plans subject to medical necessity review. For more information about Luxturna, click here.

  1. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. FDA News Release. December 19, 2017. Retrieved from: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm
  2. Personal communication from Spark Therapeutics, Philadelphia, Pennsylvania. January 3, 2018.