Spark Therapeutics (ONCE) announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, adopted a positive opinion recommending approval of Luxturna, a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. The positive CHMP opinion is based on data from a Phase 1 clinical trial, its follow-up trial and a Phase 3 trial that together enrolled 43 participants with inherited retinal disease caused by mutations on both copies of the RPE65 gene. The Phase 3 trial was the first randomized, controlled Phase 3 gene therapy trial for a genetic disease. Spark Therapeutics has received orphan product designation for Luxturna from EMA for the treatment of inherited retinal dystrophies. A marketing authorization decision from the European Commission is anticipated approximately within two months. If approved, the authorization will be valid in all 28-member states of the European Union, as well as Iceland, Liechtenstein and Norway. In January, Spark Therapeutics entered into a licensing and supply agreement with Novartis (NVS) to commercialize Luxturna when and if approved in Europe and all markets outside the U.S.
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