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The First FDA-Approved Gene Therapy Can Cure a Type of Blindness—But May Cost $1 Million

December 19, 2017, 5:12 PM UTC
Iris Network Student
PORTLAND, ME - FEBRUARY 23: Julie Voisine, 53, walks down the side of Congress Street downtown blindfolded as part of her training with her teacher, Mike Dionne, who is an orientation and mobility specialist with the Iris Network. Voisine has retinitis pigmentosa a progressive genetic eye condition that will eventually leave her totally blind. (Staff photo by Brianna Soukup/Portland Press Herald via Getty Images)
Portland Press Herald Press Herald via Getty Images

The Food and Drug Administration (FDA) on Tuesday approved the first true gene therapy in the U.S.—Luxturna from Spark Therapeutics, which treats a form of inherited vision loss which can lead to blindness. The landmark approval continues a string of significant medical advances to be blessed by regulators this year. But Spark is staying mum on Luxturna’s price so far amid speculation that the treatment’s list price could be as high as $1 million. Spark Therapeutics stock stayed relatively flat in early Tuesday trading.

Luxturna is a gene therapy that’s directly administered to patients who have a condition called biallelic RPE65 mutation-associated retinal dystrophy. The disorder is caused by defects in a gene which helps produce a protein critical to normal vision. With Luxturna, a healthy copy of the gene is inserted into retinal cells (with the help of engineered viruses) in order to produce the necessary protein and restore vision.

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The science is futuristic, and the fact the idea that gene therapies like Luxturna may be able to rectify lifelong genetic defects over the long term is tantalizing. “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb in a statement.

But groundbreaking science can come at a backbreaking price. There has been speculation, for instance, that Luxturna could cost as much as $1 million (Spark will not be revealing the list price until January). While patients wouldn’t have to pay such a price out-of-pocket, the sticker shock could prove a problem to insurers and benefits managers, and discourage widespread prescriptions by doctors.

Cutting-edge but high cost drugs that target a relatively small portion of the population have been on the rise recently and spurred debates about how the health care system should balance patient access and market incentives for innovation. Earlier this year the FDA approved two groundbreaking new types of cancer treatments—Novartis’ Kymriah and Gilead/Kite’s Yescarta—which reengineer immune cells to fight cancer but also come with list prices in the hundreds of thousands of dollars.