voretigene neparvovec-rzyl (Luxturna)
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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]
More general terms
References
- ↑ 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
- ↑ 2.0 2.1 Associated Press. Jan 3, 2018 [2]
- ↑ 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 - ↑ 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: https://www.ncbi.nlm.nih.gov/pubmed/28712537