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Category: Gene Therapy

FDA Approves the First Gene Therapy to Treat an Inherited Disease

On December 19, 2017, FDA approved Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl), the United States’ first gene therapy approved to treat an inherited genetic disease.  This approval follows that of Novartis’ Kymriah (tisagenlecleucel) and Gilead Sciences’ Yescarta (axicabtagene ciloleucel), both gene therapies approved earlier this year to treat certain forms of cancer.  Gene therapy has arrived, and although the genetic material central to these new treatments is not expressly listed in the statutory definition of “biological product,” FDA is regulating these products as biologics, giving them twelve-year non-patent exclusivity.

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