Posted by Michael Wonder on 19 May 2017
Spark Therapeutics completes rolling biologics license application submission to FDA for investigational gene therapy voretigene neparvovec
18 May 2017 - Voretigene neparvovec has the potential to be the first pharmacologic treatment for an inherited retinal disease and the first gene therapy for a genetic disease in the U.S.
Spark Therapeutics today announced the completion of the rolling submission of a biologics license application with the U.S. FDA for voretigene neparvovec, an investigational, one-time gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal disease.
The submission includes data from three clinical trials that enrolled 41 participants with RPE65-mediated inherited retinal disease, including the first randomised, controlled Phase 3 trial for a gene therapy for a genetic disease.
