Voretigene neparvovec explained

Gt Target Gene:RPE65
Gt Vector:Adeno-associated virus serotype 2
Gt Nucleic Acid Type:DNA
Tradename:Luxturna
Dailymedid:Voretigene neparvovec
Pregnancy Au:B2
Pregnancy Au Comment:[1]
Routes Of Administration:Subretinal injection
Atc Prefix:S01
Atc Suffix:XA27
Legal Au:S4
Legal Ca:Rx-only / Schedule D
Legal Ca Comment:[2] [3]
Legal Us:Rx-only
Legal Eu:Rx-only
Legal Eu Comment:[4]
Cas Number:1646819-03-5
Drugbank:DB13932
Unii:2SPI046IKD
Kegg:D11008
Synonyms:voretigene neparvovec-rzyl

Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis.[5]

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. The gene therapy is not a cure for the condition, but substantially improves vision in those treated.[6] It is given as a subretinal injection.

Voretigene neparvovec was approved for medical use in the United States in December 2017,[7] Australia in August 2020[8] and in Canada, in October 2020.[9] It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).[10]

Medical uses

Voretigene neparvovec is indicated for the treatment of people with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

Chemistry and production

Voretigene neparvovec is an AAV2 vector containing human RPE65 cDNA with a modified Kozak sequence. The virus is grown in HEK 293 cells and purified for administration.[11]

History

It was developed by Spark Therapeutics and Children's Hospital of Philadelphia.[12] [13] [14]

It was granted orphan drug designation for Leber congenital amaurosis and retinitis pigmentosa.[15] [16] A biologics license application was submitted to the US Food and Drug Administration (FDA) in July 2017 with Priority Review.[17] Phase III clinical trial results were published in August 2017.[18] On 12 October 2017, a key advisory panel to the FDA, composed of 16 experts, unanimously recommended approval of the treatment.[19] The FDA approved the drug in December 2017.[20] With the approval, Spark Therapeutics received a pediatric disease priority review voucher.[21]

The first commercial sale of voretigene neparvovec, which was also the first sale of any gene therapy product in the United States, occurred in March 2018.[22] The price of the treatment at the time was announced as being $425,000 per eye.[23]

Further reading

Notes and References

  1. Web site: Luxturna Australian Prescription Medicine Decision Summary. Therapeutic Goods Administration (TGA) . 13 August 2020 . 16 August 2020.
  2. Web site: Luxturna Product information . Health Canada . 25 April 2012 . 21 October 2020.
  3. Web site: Summary Basis of Decision (SBD) for Luxturna . Health Canada . 23 October 2014 . 29 May 2022.
  4. Web site: Luxturna EPAR . European Medicines Agency (EMA) . 24 September 2018 . 21 October 2020.
  5. Web site: Luxturna- voretigene neparvovec-rzyl kit . DailyMed . 4 December 2019 . 14 August 2020.
  6. News: McGinley L . FDA approves first gene therapy for an inherited disease. Washington Post. 19 December 2017.
  7. Web site: 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 . 28 November 2022.
  8. Web site: Luxturna . Therapeutic Goods Administration (TGA) . 13 August 2020 . 22 September 2020.
  9. Web site: 14 October 2020. 'I never saw stars before': Gene therapy brings back 8-year-old Canadian boy's sight. 21 October 2020. CTVNews.
  10. Web site: First Gene Therapy For Inherited Disease Gets FDA Approval. 19 December 2017 . NPR .
  11. Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM . 6 . Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial . Lancet . 390 . 10097 . 849–860 . August 2017 . 28712537 . 5726391 . 10.1016/S0140-6736(17)31868-8 . Janet Wittes .
  12. Web site: Spark's gene therapy for blindness is racing to a historic date with the FDA. 9 October 2017. Statnews.com. 9 October 2017.
  13. News: Gene Therapy for Blindness Appears Initially Effective, Says U.S. FDA . Clarke T . Scientific American. 12 October 2017.
  14. News: FDA approves Spark's gene therapy for rare blindness pioneered at CHOP . Philly. 24 March 2018.
  15. Web site: Voretigene neparvovec - Spark Therapeutics - AdisInsight . adisinsight.springer.com.
  16. News: FDA Panel Backs Gene Therapy for Inherited Blindness. Medscape . Lewis R . 13 October 2017.
  17. Web site: Press Release - Investors & Media - Spark Therapeutics. Ir.sparktx.com. 9 October 2017.
  18. Lee H, Lotery A . 26983863 . Gene therapy for RPE65-mediated inherited retinal dystrophy completes phase 3 . Lancet . 390 . 10097 . 823–824 . August 2017 . 28712536 . 10.1016/S0140-6736(17)31622-7 .
  19. News: Landmark Therapy to Treat Blindness Gets One Step Closer to FDA Approval. 12 October 2017. Bloomberg.com. 12 October 2017.
  20. Web site: Luxturna . U.S. Food and Drug Administration (FDA) . 19 December 2017 . 2 April 2020.
  21. Web site: Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price . FiercePharma. 19 December 2017 .
  22. News: The anxious launch of Luxturna, a gene therapy with a record sticker price. 21 March 2018. STAT. 24 March 2018.
  23. News: Tirrell M . A US drugmaker offers to cure rare blindness for $850,000. 3 January 2018. CNBC. 3 January 2018.

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