Novartis AG/Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec) has been granted marketing authorization in the EU, the first gene therapy to be approved for the treatment of retinal disease. The Pink Sheet takes a look at how this innovative one-off treatment might now get to market and how Europe’s payers might cope with the growing numbers of high-cost, game-changing therapies that are set to hit the market.
Luxturna has been approved for children and adults with inherited retinal dystrophy caused by RPE65 gene mutations. This rare genetic disorder causes vision loss and usually leads to blindness, and affects one in 200,000 people
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