The U.S. Food and Drug Administration (FDA) has approved the use of a new form of gene therapy that targets a rare inherited eye disease. The FDA panel had voted to approve the therapy back in October.
The treatment, voretigene neparvovec, will now be sold as Luxturna. It can be used to treat children and adult patients suffering from the eye disease, which can cause blindness. The disease is believed to be caused by a mutation in the RPE65 gene. Luxturna is the first drug of its kind to get FDA approval.
As explained by the FDA, the RPE65 gene contains essential instructions on how to produce the enzyme necessary for normal vision. If there’s a mutation in the RPE65 gene, it can reduce RPE65 activity, which in turn can impair a person’s vision as early as infancy.
However, researchers found that by applying Luxturna using subretinal injection, where a normal copy of the RPE65 gene is provided directly to retinal cells, the cells would begin to “produce the normal protein that converts light to an electrical signal in the retina to restore patient’s vision loss.” Treatment has to be done separately in each eye, with at least 6 days between each surgery.
“The approval of Luxturna further opens the door to the potential of gene therapies,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER) in a statement. “Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.”
The FDA explains the RPE65 mutation affects around 1,000 to 2,000 patients in the U.S, with Spark Therapeutics Inc — developers of Luxturna — previously stating nearly 6,000 people around the world could benefit from the treatment.
There are some side effects of the gene therapy, including eye redness, cataract, and retinal tear. The FDA also said Luxturna should only be used on patients if their physician can confirm they have viable retinal cells. Considering the treatment could potentially help them regain their sight, patients may be willing to take the risk.
“I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, M.D. “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening.”