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Spark's Gene Therapy Approved

The US Food and Drug Administration approved the first gene therapy that treats an inherited disease yesterday, NPR reports.

The therapy, Spark Therapeutics' Luxturna, treats retinal dystrophy, a form of childhood blindness caused by mutations in the RPE65 gene. In October, an FDA panel recommended that the treatment be approved. Two other gene therapies —Novartis' Kymriah and Kite Pharmaceutical's Yescarta — have also been approved this year, though those are both cancer treatments.

Treatment with Luxturna involves two injections, one to each eye, to deliver viruses containing normal versions of RPE65 gene. NPR writes that some patients treated with Luxturna experienced "dramatic improvements" and were able to read and play sports.

"One of the best things I've ever seen since surgery are the stars. I never knew that they were little dots that twinkled," patient Mistie Lovelace, who urged the FDA at a public meeting to approve the therapy, tells the Associated Press.

However, the treatment is likely to be expensive, with predictions placing it at $1 million, according to the AP. Spark, though, hasn't announced the cost. "I think the price tag will be enormous — 20 or 30 times the annual wages of the typical American," Peter Bach, director of the Center for Health Policy and Outcomes at the Memorial Sloan Kettering Cancer Center, tells NPR.