Spark Therapeutics presents data reinforcing efficacy of voretigene neparvovec
Spark Therapeutics announced that Principal Investigator Albert M. Maguire, M.D., professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania, presented one-year efficacy data from the crossover group and two-year durability data from the original intervention group for the Phase 3 trial of voretigene neparvovec -- formerly SPK-RPE65 -- during Retina Subspecialty Day at the American Academy of Ophthalmology 2016 Annual Meeting in Chicago. Voretigene neparvovec, an investigational gene therapy for Inherited Retinal Disease caused by mutations in the RPE65 gene, has received both breakthrough therapy and orphan product designations from the U.S. FDA, as well as orphan product designations from the EMA. The crossover group consists of nine subjects who, after one year of undergoing the same retinal and visual function testing as the original intervention group, elected to cross over and receive voretigene neparvovec in both eyes. One year after administration, the mean improvement among all nine subjects, as measured by the primary endpoint, a multi-luminance mobility test, was 2.1 lux levels. Dr. Maguire also presented data that showed that the original intervention group subjects who received voretigene neparvovec maintained improvement demonstrated at year one, as measured in MLMT and full-field light sensitivity threshold testing, for at least two years. The mean MLMT improvement of 1.9 lux levels was sustained after two years. The more than 100-fold average improvement in FST testing observed in the original intervention group at one year similarly was maintained through at least two years.