ICER will develop a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Luxturna®, 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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Below you will find the final documents from the assessment review process: