The pharmaceutical company Spark Therapeutics recently announced FDA approval1,2 of a gene therapy known as Luxturna, which has been shown to improve vision in patients with an extremely rare inherited eye disease caused by mutations in the gene RPE65. Scientists estimate this disease to occur in less than one in 500,000 people – or about 560 people total in the United States3,4.
The company has priced the drug at $425,000 per eye ($850,000 per patient), which does not include the cost of the surgery5.