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.
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
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