Data from Pharmawand - Curated by Toby Galbraith - Date added 07 August 2017

Spark Therapeutics , a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (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 randomized, controlled Phase III trial for a gene therapy for a genetic disease. Once EMA has validated the application, the review period will begin. Spark Therapeutics has previously received orphan product designations for Luxturna from EMA for the treatment of both Leber congenital amaurosis and retinitis pigmentosa.


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