Dec 19 (Reuters) - The U.S. Food and Drug Administration on Tuesday approved Spark Therapeutics Inc’s treatment for a rare form of blindness, marking the first time the agency has approved a gene therapy for an inherited disease.
The drug, Luxturna, treats inherited retinal disease caused by defects in a gene known as RPE65, which tells cells to produce an enzyme critical for normal vision. The condition affects between 1,000 and 2,000 people in the United States.
Luxturna works by delivering 150 billion viral vector particles containing a correct copy of the RP65 gene to retinal cells, restoring their ability to make the needed enzyme. It is given just once. (Reporting by Toni Clarke in Washington Editing by Chizu Nomiyama)