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  • By Anni Griswold
    Spark Therapeutics, Forbes
    Comprehensive Ophthalmology, Pediatric Ophth/Strabismus, Retina/Vitreous

    The cost to restore sight to the blind will be a “responsible” $425,000 per eye, Spark Therapeutics announced this week. The company set the price of Luxturna and layed out options for insurers and patients to deal with the financial burden.

    Luxturna, the first gene therapy approved for an inherited disease, gained regulatory approval on December 19. A single dose swaps out defective copies of the RPE65 gene, a major cause of Leber congenital amaurosis and other retinal dystrophies, for functional versions. An estimated 6,000 patients worldwide and about 2,000 individuals in the United States could benefit from the treatment.

    “Many were anticipating this was going to be over a million dollars because it’s a small patient population,” Steve Miller, the chief medical officer of Express Scripts, told Forbes. “To be very frank, they’ve hit on a responsible price. Is it inexpensive? Absolutely not. But it’s responsible.”

    Insurers and government agencies are expected to pick up the brunt of the bill, lightening the financial burden on individual patients. Spark Therapeutics has reached tentative agreements with 2 healthcare insurers, Harvard Pilgrim and Express Scripts, to limit out-of-pocket costs to patients. The company may offer additional rebates to government and private insurers if clinical outcomes do not meet expectations.

    The drug has shown promising efficacy in clinical trials. A single subretinal injection of Luxturna improved light perception for at least 3 years in affected children and adults. Trial participants continue to be followed for long-term outcomes.