FDA approves gene therapy in fight against blindness

In a decision that accelerates a new era for gene therapy, the Food and Drug Administration on Tuesday approved a DNA-altering medication that can reverse an inherited form of progressive blindness.

The FDA’s blessing makes Luxturna, a treatment for retinal dystrophy caused by a specific genetic mutation, the first gene therapy cleared for use in the U.S. to treat a genetic disease other than cancer.

Tuesday’s announcement marks the third time in five months that the drug safety agency has allowed a gene therapy—a form of treatment with a long and fitful safety history—on the U.S. market. The first approval went to Kymriah, which treats a form of leukemia, in August. In October, the drug agency cleared a second gene-based treatment called Yescarta to treat a form of lymphoma.

“Gene therapy will become a mainstay in treating, and maybe curing, many of our most devas-

tating and intractable illnesses,” Dr. Scott Gottlieb, the FDA’s commissioner, said Tuesday. “We’re at a turning point when it comes to this novel form of therapy.”

The agency will release guidelines in the coming year to speed the evaluation and approval of additional gene therapy treatments for a range of diseases so that the country may “capitalize on this scientific opening,” he added. Among other things, the guidelines will include new ways to measure whether a gene therapy is working.

The newest gene therapy, known to scientists as voretigene neparvovec-rzyl, will be used to treat children and adults with an inherited form of vision loss. It often begins in childhood or adolescence and progresses to complete blindness in adulthood.

Between 1,000 and 2,000 people in the United States have inherited two faulty copies of the RPE65 gene, one from each parent. Luxturna replaces those faulty genes with normal versions, thus erasing the mutations’ harmful effects.

Luxturna’s safety was established in a clinical development program that tested the treatment on 41 patients between the ages of 4 and 44, the FDA said. Among the most common side effects were eye redness, cataracts, increased pressure within the eye and retinal tears.

In a yearlong clinical trial with 31 subjects affected by this form of retinal dystrophy, those treated with Luxturna showed greater improvements in their ability to navigate an obstacle course in low light compared with untreated subjects in the control group. Treatment with Luxturna must be done separately in each eye on separate days.