The FDA Just Approved a Game-Changing Therapy for Blindness

The FDA Just Approved a Game-Changing Therapy for Blindness

A decision on Spark Therapeutics' (NASDAQ: ONCE) vision-restoring gene therapy, Luxturna, was expected next month, but the Food and Drug Administration decided an early OK was warranted following a unanimous recommendation by its advisory committee in October. The approval is a big breakthrough for patients and the company, but Luxturna's price could raise some eyebrows. 

Luxturna is a gene therapy that can restore functional vision in 90% of patients with biallelic RPE65 mutation-associated retinal dystrophy. Patients with this rare condition lose their vision because they fail to produce an adequate amount of RPE65, a protein that's critical to sight. Typically, patients with this genetic mutation lose their vision over time and many of these patients eventually become completely blind. There are approximately 1,000 to 2,000 patients in the U.S. with biallelic RPE65 mutation-associated retinal dystrophy.

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Source: Fool.com