The FDA announced the approval of Luxturna to treat children and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
Luxturna (voretigene neparvovec, Spark Therapeutics) is the first directly administered gene therapy approved in the United States to target a disease caused by mutations in a specific gene, according to an FDA news release.
“The approval today of the first directly administered gene therapy is especially notable, not only for what the new treatment does and how it works, by engineering a virus as a vehicle to deliver the gene directly to a target inside the body, but also for how we’ve expanded the use of gene therapy beyond the treatment of cancer to the treatment of vision loss for children and adults,” FDA Commissioner Scott Gottlieb, MD, said in a media briefing.
Gottlieb announced the FDA will next year issue a suite of disease-specific guidance documents on the development of gene therapy products as part of a “modern comprehensive framework for how we’ll help advance the field of gene therapy.”
Luxturna delivers a normal copy of the RPE65 gene to retinal cells, which produce the normal protein that converts light to an electrical signal in the retina to restore the patient’s vision. A naturally occurring adeno-associated virus serves as a vehicle to deliver the normal RPE65 gene to the retinal cells, according to the release.
Biallelic RPE65 mutation-associated retinal dystrophy affects 1,000 to 2,000 patients in the U.S., according to the FDA.
The safety and efficacy of the therapy were established in a clinical development program with 41 patients, each with confirmed biallelic RPE65 mutations. The primary efficacy evidence was based on a phase 3 study of 31 patients. The patients who received Luxturna demonstrated significant improvements in their ability to complete an obstacle course at low light levels when compared with a control group, according to the FDA.
“In contrast to previous approvals, which refer to what we are essentially calling personalized cell-based gene therapy products, today’s approval is for a gene therapy product that can be given directly to an individual to treat a disease,” Peter Marks, MD, PhD, director of the FDA Center for Biologics Evaluation and Research, said at the briefing. – by Robert Linnehan
Editor’s note: This article has been updated to include remarks made at a media briefing by FDA Commissioner Scott Gottlieb, MD, and FDA Center for Biologics Evaluation and Research Director Peter Marks, MD, PhD.