Spark Therapeutics has submitted is seeking European marketing approval for Luxtuna – the proposed trade name for voretigene neparvovec.
Voretigene neparvovec is 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 Marketing Authorization Application includes data from three clinical trials that enrolled 41 participants with RPE65-mediated inherited retinal dystrophy, including the first randomised, controlled Phase 3 trial for a gene therapy for a genetic disease.
Once the European Medicines Agency (EMA) has validated the application, the review period will begin.
Spark Therapeutics has previously received orphan product designations for Luxtuna from EMA for the treatment of both Leber congenital amaurosis and retinitis pigmentosa.
Elsewhere, the investigational therapy is currently under Priority Review with the US Food and Drug Administration.
“With Luxtuna now in regulatory review on both sides of the Atlantic, we are building out our medical and commercial infrastructure to prepare to bring investigational Luxtuna to patients in the U.S. and Europe,” said John Furey, COO of Spark Therapeutics.