Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis.[4]
Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited disorder causing progressive blindness. Voretigene is the first treatment available for this condition.[7] The gene therapy is not a cure for the condition, but substantially improves vision in those treated.[8] It is given as a subretinal injection.
Voretigene neparvovec was approved for medical use in the United States in December 2017,[9] Australia in August 2020[10] and in Canada, in October 2020.[11] It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).[12]
^ ab"Luxturna Australian Prescription Medicine Decision Summary". Therapeutic Goods Administration (TGA). 13 August 2020. Retrieved 16 August 2020.
^"Luxturna Product information". Health Canada. 25 April 2012. Retrieved 21 October 2020.
^"Summary Basis of Decision (SBD) for Luxturna". Health Canada. 23 October 2014. Retrieved 29 May 2022.
^ ab"Luxturna- voretigene neparvovec-rzyl kit". DailyMed. 4 December 2019. Retrieved 14 August 2020.
^Cite error: The named reference FDA Luxturna was invoked but never defined (see the help page).
^Cite error: The named reference sparktx.com was invoked but never defined (see the help page).
^McGinley L (19 December 2017). "FDA approves first gene therapy for an inherited disease". Washington Post.
^"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss". U.S. Food and Drug Administration (FDA). 24 March 2020. Retrieved 28 November 2022.
^"Luxturna". Therapeutic Goods Administration (TGA). 13 August 2020. Retrieved 22 September 2020.
^"'I never saw stars before': Gene therapy brings back 8-year-old Canadian boy's sight". CTVNews. 14 October 2020. Retrieved 21 October 2020.
^"First Gene Therapy For Inherited Disease Gets FDA Approval". NPR. 19 December 2017.
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