Voretigene neparvovec

Voretigene neparvovec, sold under the brand name Luxturna, is a novel gene therapy for the treatment of Leber congenital amaurosis.[1] It was developed by Spark Therapeutics and Children's Hospital of Philadelphia.[2][3] It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).[4]

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
  • US: N (Not classified yet)
    Routes of
    administration    		Subretinal injection
    ATC code
    Legal status
    Legal status
    Identifiers
    CAS Number
    DrugBank
    UNII
    KEGG

    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.[5] The gene therapy is not a cure for the condition, but substantially improves vision in those treated.[6] 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.[7]

    History

    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.[8]

    It was granted orphan drug designation for Leber congenital amaurosis and retinitis pigmentosa.[9][10] A biologics license application was submitted to the FDA in July 2017 with Priority Review.[5] 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.[13] With the approval, Spark Therapeutics received a pediatric disease priority review voucher.[14]

    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.[15][15][4] The price of the treatment at the time was announced as being $425,000 per eye.[16]

    gollark: You should probably not share the repo where people can see it.
    gollark: * Divergent Naur form
    gollark: They have some kind of convoluted lexer thing instead of my shiny 26 lines of parser combinators.
    gollark: This is VERY convoluted.
    gollark: Ask them to send me their interpreter so I can observe it.

    References
    1. "Luxturna- voretigene neparvovec-rzyl kit". DailyMed. 4 December 2019. Retrieved 14 August 2020.
    2. "Spark's gene therapy for blindness is racing to a historic date with the FDA". Statnews.com. 9 October 2017. Retrieved 9 October 2017.
    3. Clarke T. "Gene Therapy for Blindness Appears Initially Effective, Says U.S. FDA". Scientific American. Retrieved 12 October 2017.
    4. "First Gene Therapy For Inherited Disease Gets FDA Approval". NPR. 19 December 2017.
    5. "Press Release - Investors & Media - Spark Therapeutics". Ir.sparktx.com. Retrieved 9 October 2017.
    6. McGinley L (19 December 2017). "FDA approves first gene therapy for an inherited disease". Washington Post.
    7. Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. (August 2017). "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. doi:10.1016/S0140-6736(17)31868-8. PMC 5726391. PMID 28712537.
    8. "FDA approves Spark's gene therapy for rare blindness pioneered at CHOP - Philly". Philly.com. Retrieved 24 March 2018.
    9. "Voretigene neparvovec - Spark Therapeutics - AdisInsight". adisinsight.springer.com.
    10. Lewis R (13 October 2017). "FDA Panel Backs Gene Therapy for Inherited Blindness". Medscape.
    11. Lee H, Lotery A (August 2017). "Gene therapy for RPE65-mediated inherited retinal dystrophy completes phase 3". Lancet. 390 (10097): 823–824. doi:10.1016/S0140-6736(17)31622-7. PMID 28712536. S2CID 26983863.
    12. "Landmark Therapy to Treat Blindness Gets One Step Closer to FDA Approval". Bloomberg.com. 12 October 2017. Retrieved 12 October 2017.
    13. "Luxturna". U.S. Food and Drug Administration (FDA). 19 December 2017. Retrieved 2 April 2020.
    14. "Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price". FiercePharma.
    15. "The anxious launch of Luxturna, a gene therapy with a record sticker price". STAT. 21 March 2018. Retrieved 24 March 2018.
    16. Tirrell M (3 January 2018). "A US drugmaker offers to cure rare blindness for $850,000". CNBC. Retrieved 3 January 2018.

    Further reading
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