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Spark Therapeutics' gene therapy for a rare blindness gets $850K price tag

Gene therapy for rare blindness

WASHINGTON— A first-of-its kind genetic treatment for blindness will cost $850,000. While it's less than the $1 million price tag that analysts had expected, it's still among the most expensive genetic therapies in the world.

Spark Therapeutics says it decided on the lower price tag for Luxturna after hearing from health insurers about their ability to cover the injectable treatment. Consternation over skyrocketing drug prices in the U.S. has led to intense scrutiny from patients, Congress, insurers and hospitals.

Luxturna, approved last month by the Food and Drug Administration, is the nation's first gene therapy for an inherited disease. It can improve the vision of those with a rare form of blindness that affects just a few thousand people in the U.S. 

Patients with the condition generally start losing their sight before 18, almost always progressing to total blindness. 

The defective gene that causes the disease can be passed down for generations undetected before suddenly appearing when a child inherits a copy from both parents. 

Due to the rarity of the condition it treats, the therapy has only been tested on fewer than 50 people. The company's CEO, however, has said the effects of treatment could be "life-long."

The FDA has given the green light to several gene therapies since last summer, as decades of research into the genetic building blocks of life begin translating into marketable treatments. 

The previous two are custom-made treatments for forms of blood cancer. Novartis' Kymriah is priced at $475,000 for a one-time infusion of genetically enhanced cells. Gilead Sciences' similar treatment, Yescarta, costs $373,000 per treatment.

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