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Spark's Early Christmas Present: US FDA Approves Luxturna For Vision Loss

 
• By Sue Sutter

Review and approval of first directly administered gene therapy targeting a disease caused by genetic mutations embodies 'spirit' of agency's forthcoming series of guidance documents on gene therapies, FDA Commissioner Gottlieb says; documents are likely to encourage development of the kind of novel and clinically meaningful endpoint that Spark used in its clinical program.

Luxturna (voretigene neparvovec-rzyl

The US FDA's early approval of Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec-rzyl) for inherited vision loss – the third gene therapy licensed in 2017 – created an opening for Commissioner Scott Gottlieb to highlight the agency's plans in the coming year to further align its policy framework with the burgeoning scientific field.

On Dec

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US FDA Advisory Committee Misperceptions Abound … At HHS

 
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