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Voretigene neparvovec

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Voretigene neparvovec
Gene therapy
VectorAdeno-associated virus serotype 2
Nucleic acid typeDNA
Editing methodRPE65
Clinical data
Trade namesLuxturna
Routes of
administration		subretinal injection
ATC code
Legal status
Legal status

Voretigene neparvovec (Luxturna) is a novel gene therapy for the treatment of Leber's congenital amaurosis.[1] Married researchers Jean Bennett and Albert Maguire, among others, worked for decades on studies of congenital blindness, culminating in approval of a novel therapy, Luxturna.[2] It was developed by Spark Therapeutics and Children's Hospital of Philadelphia.[3][4] It is the first in vivo gene therapy approved by the FDA.[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.[6] The gene therapy is not a cure for the condition, but substantially improves vision in those treated.[7] It is given as an subretinal injection.

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.[8]

History

It was granted orphan drug status for Leber congenital amaurosis and retinitis pigmentosa.[9][10] A biologics license application was submitted to the FDA in July 2017 with Priority Review.[6] Phase III clinical trial results were published in August 2017.[11] On 12 October 2017, a key advisory panel to the Food and Drug Administration (FDA), composed of 16 experts, unanimously recommended approval of the treatment.[12] The US FDA approved the drug on December 19, 2017. With the approval, Spark Therapeutics received a pediatric disease priority review voucher.[13]

The first commercial sale of voretigene neparvovec -- the first for any gene therapy product in the US -- occurred in March 2018.[14][14][5] The price of the treatment has been announced at $425,000 per eye.[15]

References

  1. ^ "Luxturna (voretigene neparvovec-rzyl) label" (PDF). FDA. December 2017. Retrieved 31 December 2017. (for label updates, see FDA index page)
  2. ^ "FDA approves Spark's gene therapy for rare blindness pioneered at CHOP - Philly". Philly.com. Retrieved 2018-03-24.
  3. ^ "Spark's gene therapy for blindness is racing to a historic date with the FDA". Statnews.com. 9 October 2017. Retrieved 9 October 2017.
  4. ^ Clarke,Reuters, Toni. "Gene Therapy for Blindness Appears Initially Effective, Says U.S. FDA". Scientific American. Retrieved 2017-10-12. {{cite news}}: |last= has generic name (help)
  5. ^ a b "First Gene Therapy For Inherited Disease Gets FDA Approval". NPR.org. 19 Dec 2017.{{cite web}}: CS1 maint: date and year (link)
  6. ^ a b "Press Release - Investors & Media - Spark Therapeutics". Ir.sparktx.com. Retrieved 9 October 2017.
  7. ^ McGinley, Laurie (19 December 2017). "FDA approves first gene therapy for an inherited disease". Washington Post.
  8. ^ 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 voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, 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.
  9. ^ "Voretigene neparvovec - Spark Therapeutics - AdisInsight". adisinsight.springer.com.
  10. ^ Ricki Lewis, PhD (October 13, 2017). "FDA Panel Backs Gene Therapy for Inherited Blindness". Medscape.
  11. ^ Lee, Helena; Lotery, Andrew (26 August 2017). "Gene therapy for RPE65-mediated inherited retinal dystrophy completes 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.
  12. ^ "Landmark Therapy to Treat Blindness Gets One Step Closer to FDA Approval". Bloomberg.com. 2017-10-12. Retrieved 2017-10-12.
  13. ^ "Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price | FiercePharma". www.fiercepharma.com.
  14. ^ a b "The anxious launch of Luxturna, a gene therapy with a record sticker price". STAT. 2018-03-21. Retrieved 2018-03-24.
  15. ^ Tirrell, Meg (3 January 2018). "A US drugmaker offers to cure rare blindness for $850,000". CNBC. Retrieved 3 January 2018.
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