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Voretigene neparvovec receives rare pediatric disease designation from FDA
The FDA offices of Orphan Products Development and Pediatric Therapeutics have designated voretigene neparvovec as a drug for a rare pediatric disease, according to a Spark Therapeutics press release.Voretigene neparvovec, which will be marketed under the name Luxturna, could be both the first gene therapy for a genetic disease and the first pharmacologic treatment for an inherited retinal disease in the United States, according to the release. It is being reviewed for the treatment of biallelic RPE65 mutation-associated retinal dystrophy. A biologics license application for Luxturna was recently accepted by (Read more...)