Posted by Michael Wonder on 01 Aug 2017
Spark Therapeutics submits marketing authorisation application to European Medicines Agency for investigational Luxturna (voretigene neparvovec)
31 August 2017 - Potential for first gene therapy for a genetic disease to be approved in both the U.S. and EU.
Spark Therapeutics announced today that it has submitted a marketing authorisation application to the EMA for Luxturna, the proposed trade name for voretigene neparvovec, an investigational, one-time gene therapy for the treatment of patients with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations.
The MAA includes data from three clinical trials that enrolled 41 participants with RPE65-mediated inherited retinal dystrophy (IRD), including the first randomised, controlled Phase 3 trial for a gene therapy for a genetic disease.
