FLICKR, HELGA BIRNA JÓNASDÓTTIRUpdate (December 19): The US Food and Drug Administration (FDA) today approved Luxturna for the treatment of an inherited form of vision loss. It is the first gene therapy approved in the U.S. to correct a specific disease-linked gene. “We’re at a turning point when it comes to this novel form of therapy,” FDA Commissioner Scott Gottlieb said in a statement. “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”
A panel of 16 independent advisers to the US Food and Drug Administration (FDA) voted unanimously today (October 12) to recommend the approval of Luxturna, a gene therapy meant to treat Leber congenital amaurosis—a rare, inherited form of childhood blindness—and other retinal disorders, STAT News reports. If the agency agrees with the panel’s recommendation, Luxturna would be the first gene therapy aimed at correcting a congenital defect approved in the U.S.
The therapy, developed by Philadelphia-based Spark Therapeutics, is injected directly into the eye in a one-time treatment. Billions of virus particles deliver a functional copy of the RPE65 gene—which encodes a protein necessary for normal vision and is mutated in patients with certain vision disorders—to the retina. So far, more than two dozen children ...
