Voretigene neparvovec


Voretigene neparvovec ( Luxturna ) is a novel gene therapy for the treatment of Leber’s congenital amaurosis . It was developed by Spark Therapeutics and Children’s Hospital of Philadelphia . [1] [2] It is the first in vivo gene therapy approved by the FDA. [3]

Leber’s congenital amaurosis, or biallelic RPE65- mediated inherited retinal disease, is an inherited progressive progressive blindness. Voretigene is the first treatment for this condition. [4]The gene therapy is not a cure for the condition, but rather improves vision in those treated. [5] It is given as an subretinal injection.

The price of the treatment has not been announced, but it is expected to cost around $ 1 million. [3] [6]

Development

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. [7]

It was granted orphan drug status for Leber congenital amaurosis and retinitis pigmentosa . [8] [ better source needed ] A biologics license application was submitted to the FDA in July 2017 with Priority Review . [4] Phase III clinical trial results were published in August 2017. [9] On October 12, 2017, a key advisory panel to the Food and Drug Administration (FDA) , composed of 16 experts, unanimously recommended approval of the treatment. [10] The US FDA approved the drug on December 19, 2017. With the approval, Spark Therapeutics received apediatric disease priority review voucher . [11]

References

  1. Jump up^ “Spark’s gene therapy for blindness is racing to a historic date with the FDA” . Statnews.com . October 9, 2017 . Retrieved 9 October 2017 .
  2. Jump up^ Clarke, Reuters, Toni. “Gene Therapy for Blindness Initially Effective Appears, US Says FDA” . Scientific American . Retrieved 2017-10-12 .
  3. ^ Jump up to:b “First Gene Therapy For Inherited Disease Gets FDA Approval” . NPR.org . Dec 19, 2017.
  4. ^ Jump up to:b “Press Release – Investors & Media – Spark Therapeutics” . Ir.sparktx.com . Retrieved 9 October 2017 .
  5. Jump up^ McGinley, Laurie (19 December 2017). “FDA approves first gene therapy for an inherited disease” . Washington Post .
  6. Jump up^ “The First FDA-Approved Gene Therapy Can Cure a Type of Blindness-But May Cost $ 1 Million” . Fortune .
  7. Jump up^ Russell, Stephen; Bennett, Jean; Wellman, Jennifer A .; Chung, Daniel C .; Yu, Zi-Fan; Tillman, Amy; Wittes, Janet; Pappas, Julie; Elci, Okan (2017-08-26). “Efficacy and safety of neparvovec voretigene (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomized, controlled, open-label, phase 3 trial . ” The Lancet . 390 (10097): 849-860. doi : 10.1016 / S0140-6736 (17) 31868-8 . ISSN  0140-6736 . PMID  28712537 .
  8. Jump up^ “Voretigene neparvovec – Spark Therapeutics – AdisInsight” . adisinsight.springer.com .
  9. Jump up^ Lee, Helena; Lotery, Andrew (26 August 2017). “Gene therapy for RPE65-mediated inherited retinal dystrophy complete phase 3” . The Lancet . 390(10097): 823-824. doi : 10.1016 / S0140-6736 (17) 31622-7 . PMID  28712536 . Retrieved 9 October 2017 – via www.thelancet.com.
  10. Jump up^ “Landmark Therapy to Treat Blindness Gets One Step Closer to FDA Approval” . Bloomberg.com . 2017-10-12 . Retrieved 2017-10-12 .
  11. Jump up^ “Spark grabs FDA nod for Luxturna, a breakthrough gene therapy . www.fiercepharma.com .

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