Voretigene neparvovec

Voretigene neparvovec
Gene therapy
Target geneRPE65
VectorAdeno-associated virus serotype 2
Nucleic acid typeDNA
Clinical data
Trade namesLuxturna
Other namesvoretigene neparvovec-rzyl
AHFS/Drugs.comProfessional Drug Facts
License data
Pregnancy
category
Routes of
administration		Subretinal injection
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG

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] in Australia in August 2020,[10] in Canada in October 2020,[11] and in Switzerland in February 2020.[12] It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).[13]

  1. ^ a b "Luxturna Australian Prescription Medicine Decision Summary". Therapeutic Goods Administration (TGA). 13 August 2020. Retrieved 16 August 2020.
  2. ^ "Luxturna Product information". Health Canada. 25 April 2012. Retrieved 21 October 2020.
  3. ^ "Summary Basis of Decision (SBD) for Luxturna". Health Canada. 23 October 2014. Retrieved 29 May 2022.
  4. ^ a b "Luxturna- voretigene neparvovec-rzyl kit". DailyMed. 4 December 2019. Retrieved 14 August 2020.
  5. ^ Cite error: The named reference FDA Luxturna was invoked but never defined (see the help page).
  6. ^ "Luxturna EPAR". European Medicines Agency (EMA). 24 September 2018. Retrieved 21 October 2020.
  7. ^ Cite error: The named reference sparktx.com was invoked but never defined (see the help page).
  8. ^ McGinley L (19 December 2017). "FDA approves first gene therapy for an inherited disease". Washington Post.
  9. ^ "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. Archived from the original on 26 May 2019. Retrieved 28 November 2022.
  10. ^ "Luxturna". Therapeutic Goods Administration (TGA). 13 August 2020. Retrieved 22 September 2020.
  11. ^ "'I never saw stars before': Gene therapy brings back 8-year-old Canadian boy's sight". CTVNews. 14 October 2020. Archived from the original on 20 October 2020. Retrieved 21 October 2020.
  12. ^ "Luxturna (active substance: voretigene neparvovec)". Swissmedic. Retrieved 14 January 2025.
  13. ^ "First Gene Therapy For Inherited Disease Gets FDA Approval". NPR. 19 December 2017.
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