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
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Courtyard Marriott at Briarcliff
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The Midwest CEPAC convened to deliberate on ICER's report on voretigene neparvovec (LuxturnaTM, Spark Therapeutics).