FDA approves gene therapy to treat an inherited form of blindness

The FDA approved Luxturna, a first-of-its-kind gene therapy, to treat a rare form of inherited vision loss that can result in complete blindness.

Luxturna, developed by Spark Therapeutics, represents the first gene therapy for treatment of a genetic disease  in the U.S. It is approved to treat children and adults with Biallelic RPE65 mutation-associated retinal dystrophy.

"Today's approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases," said FDA Commissioner Scott Gottlieb, M.D.

Although Spark Therapeutics has yet to announce the price of the novel drug, analysts are predicting that the treatment will cost $500,000 per eye, or $1 million per patient, according to The Washington Post.

The therapy works by injecting copies of a normal version of the gene RPE65 into a patient's eye. That gene is responsible for producing a protein integral to maintaining light receptors. Patients who have the rare genetic disease have a mutated version of RPE65, which slowly causes vision loss and in most cases, almost all end up completely blind.

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