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FDA Approves First Gene Therapy for Rare Inherited Eye Disorders

December 21, 2017

The Food and Drug Administration (FDA) has approved Luxturna (voretigene neparvovec-rzyl; Spark Therapeutics Inc.) intraocular suspension for subretinal injection, the first directly administered gene therapy that targets a disease caused by mutations in a specific gene.

Luxturna is a live, non-replicating adeno-associated virus serotype 2 which has been genetically modified to express the human RPE65 gene. It is designed to deliver a normal copy of the gene encoding the human retinal pigment epithelial 65 kDa protein (RPE65) to cells of the retina in persons with reduced or absent levels of biologically active RPE65. Luxturna is indicated for the treatment of patients with confirmed biallelic RPE65 gene mutation-associated retinal dystrophy, a group of rare, inherited eye disorders that lead to vision loss and may cause complete blindness in certain patients. Luxturna should be given only to patients who have viable retinal cells, as determined by the treating physician.

FDA approval for Luxturna was based primarily on a phase III U.S. pivotal trial, which was published this year in the journal Lancet (Russell et al., 2017). The approval follows a favorable recommendation from an FDA Advisory Committee that met in October 2017 to review the Luxturna Biologics License Application.


A Hayes Prognosis Overview on Luxturna is available here.


Food and Drug Administration (FDA). FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss [news release]. December 19, 2017. Available at:

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. Accessed December 20, 2017.