Publications/White Papers

FDA approves gene therapy for rare form of vision loss

The U.S. Food and Drug Administration (FDA) has approved the use of Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl) for the treatment of a rare inherited form of vision loss and blindness. This marks the first approval by the FDA of a directly administered gene therapy for the treatment of a disease caused by mutations in a specific gene. The approval came earlier than expected, as the scheduled Prescription Drug User Fee Act (PDUFA) date chosen for the Luxturna decision was January 12, 2018. (December 19, 2017)

P4A thoughts

  • The previously-approved gene therapies (per the FDA’s broad definition) were CAR-T cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), marketed by Novartis and Gilead respectively. Most consider these to be ‘cell therapies’ as opposed to gene therapies, meaning that this approval is more important as it is the first one given for a “true” gene therapy
  • The FDA advisory committee voted unanimously for the approval of Luxturna. This provides further evidence that the FDA is fully behind the development of gene therapies, and are very keen to integrate them into the treatment landscape.
  • The FDA is taking the gene therapy revolution extremely seriously, and is spending time and resources trying to prepare the U.S. regulatory system for this technology. The FDA Commissioner, Scott Gottlieb, stated in the press release of this approval that the FDA was planning to issue “disease-specific guidance documents on the development of specific gene therapy products” in 2018 which will include “more efficient parameters…for the evaluation and review of gene therapy”.
  • Spark submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the summer of 2017. The decision from the EMA is expected at some point in early 2018.
  • Based on the strong positive opinion that Luxturna received from the FDA, it is thought that the EMA will follow suit and grant Luxturna approval in the E.U. This is further supported by the fact that the EMA has previously given approvals for other true gene therapies, including Glybera (alipogene tiparvovec) and Strimvelis, marketed by UniQure and GSK respectively

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