Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis.
Voretigene neparvovec was approved for medical use in the United States in December 2017,[9] in Australia in August 2020,[10] in Canada in October 2020,[11] and in Switzerland in February 2020.[12].
[15][16][17] It was granted orphan drug designation for Leber congenital amaurosis and retinitis pigmentosa.
[18][19] A biologics license application was submitted to the US Food and Drug Administration (FDA) in July 2017 with Priority Review.
[20] On 12 October 2017, a key advisory panel to the FDA, composed of 16 experts, unanimously recommended approval of the treatment.