The Food and Drug Administration Tuesday approved the first gene therapy to treat an inherited disease.
The treatment is called Luxturna, a genetically modified virus that ferries a healthy gene into the eyes of patients born with retinal dystrophy, a rare condition that destroys cells in the retina needed for healthy vision.
In tests on patients, the treatment often produced dramatic results, restoring the ability of patients to see things they could never see before, such as the stars, the moon, fireworks and their parents’ faces.
The treatment also enabled patients to do many things that had been impossible, such as read, play sports, ride bicycles and go outside at night by themselves.
“Today’s approval marks another first in the field of gene therapy,” said FDA Commission Scott Gottlieb in a statement announcing the decision. “This milestone reinforces the potential of this breakthrough approach in treating a wide range of challenging diseases.”