FDA Approves First Gene Therapy for Inherited Retinal Dystrophy

The U.S. Food and Drug Administration (FDA) has approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy developed by Spark Therapeutics, to treat an inherited form of vision loss that can result in blindness.  The most prevalent inherited retinal dystrophies are retinitis pigmentosa (RP), Leber congenital amaurosis (LCA) and Stargardt disease (SD), all of which have in common a mutation of the RPE65 gene. Patients with Leber congenital amaurosis will be the first beneficiaries of retinal gene therapy, with RP and SD to follow after further study.
Ocular injection of Luxturna introduces a benign virus to deliver healthy copies of the gene to the retina. In the recently-completed Spark study at the University of Iowa and Children’s Hospital of Philadelphia, patients receiving Luxturna showed significant vision improvement. This success resulted in Priority Review and Breakthrough Therapy designations from the FDA, which helps speed promising treatments through trials.
Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene. It is approved for treatment of patients with confirmed mutation of both copies of the RPE65 gene. The therapy is expected to be available in selected centers by Spring of 2018, with the price for a single dose set at at $425,000 per eye, or $850,000 per patient.
“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.
Source: FDA Press Release