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voretigene neparvovec-rzyl (Luxturna)

Indications: - biallelic RPE65 mutation-associated retinal dystrophy Dosage: - one intraocular injection each eye (once) - 5 x 10E11 vector genomes by subretinal injection in a total volume of 0.3 mL. Notes: - cost $425,000/injection ($850,000 both eyes) - Manufacturer (Swiss company Spark) promises to reimburse patients if not effective [2]

General

gene therapy

References

  1. FDA News Release. Dec 19, 2017 FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm
  2. Associated Press. Jan 3, 2018 [2]
  3. Windle ML. FDA New Drug and Biologic Approvals -- 2017 Year-in-Review Medscape - Jan 11, 2018. https://reference.medscape.com/viewarticle/890871 - U.S. Food and Drug Administration. Novel Drug Approvals for 2017. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm
  4. Russell S, Bennett J, Wellman JA et al 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. 2017 Aug 26;390(10097):849-860. Epub 2017 Jul 14. PMID: 28712537