ICER will develop a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (LuxturnaTM, Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease. Voretigene neparvovec is the first gene therapy to be approved by the US Food and Drug Administration (FDA).
Date of review: January 2018
Courtyard Marriott at Briarcliff
4000 North Mulberry Drive
Kansas City, MO
The Midwest CEPAC convened to deliberate on ICER's report on voretigene neparvovec (LuxturnaTM, Spark Therapeutics).