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FDA Panel Urges Approval Of Gene-Therapy For Genetic Form Of Blindness

October 12, 2017
An FDA expert panel unanimously voted Thursday (Oct. 12) to recommend approval of Spark Therapeutics' Luxturna (voretigene neparvovec) -- a gene therapy used to treat patients with a rare form of genetically-inherited blindness -- that some analysts say could cost nearly $1 million per patient. While the data presented did show a statistically significant improvement in the primary endpoint, FDA said it was unclear whether the endpoint -- improvement in navigating a maze -- correlated to improvements in functioning beyond...

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