Here come more gene therapies -- and more pricing debates

Forbes

11 October 2017 - Tomorrow an advisory committee to the FDA will meet to discuss Luxturna (voretigene neparvovec), a gene therapy from Spark Therapeutics to treat a rare inherited form of blindness called biallelic RPE65 mutation-associated retinal dystrophy. 

The company has not yet announced pricing for the highly anticipated product, but analysts are estimating the treatment will cost around $1 million.

If Spark wins approval for Luxturna as expected, it will inevitably add fuel to a pricing debate that flared up, most recently, in August with the FDA approval of Novartis’ Kymriah, a gene therapy for some patients with lymphoma. Novartis priced Kymriah at $475,000, which was less than many analysts expected but still enough to draw ire from folks like Steve Miller, chief medical officer of pharmacy benefits manager Express Scripts. He saw that price and declared in a blog post the need for a “new payment model” to prevent gene therapies from putting undue economic stress on the health care system.

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Michael Wonder

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Michael Wonder

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US , Pricing , Gene therapy