European Commission approves head Novartis Luxturna for treatment of hereditary retinal disease

, SwissPharmaceuticals(http://giant Novartis announced that the European Commission (EC) has approved Luxturna (voretigene neparvoc) to restore and improve vision for children and adults who have lost vision due to a double copy of the RPE65 gene mutation but retain a sufficient number of living retinal cellsapproval, making Luxturna the first gene therapy approved in Europe to treat hereditary retinal disease (IRD)LuxturnaLuxturna was developed by Spark(http://, and Novartis signed a licensing agreement with Spark in January to acquire exclusive rights to Luxturna in markets outside the United StatesSpark Therapeutics reserves the right to develop and commercialize the U.SmarketIn the U.S., Luxturna was approved by theFDA(http://in December 2017, marking the first true genetic therapy in the U.Smarket, marking the beginning of the era of gene therapychildren born with a double-copy OF the RPE65 gene mutation experience severe vision impairment from an early age, and most patients progress to complete blindnessStudies have shown that visual impairment and blindness in children often lead to social isolation, emotional distress, loss of independence, or risks such as falls and injuriesLuxturna uses adeno-related virus (AAV) technology to direct the working copy of the RPE65 gene into retinal cells to produce normal RPE65 enzymes, helping to restore vision and improve vision in children and adult patients with a sufficient number of living retinal cellsThe approval of theLuxturna is based on data from a Phase I clinical study and its follow-up study and a Phase III clinical studyIn phase 3 clinicaltrial(http://, patients with double-copy RPE65 gene mutations were able to record vision improvements as early as 30 days after receiving Luxturna treatmentin 1 year, the Luxturna treatment group's binocular multi-brightness migration rate test (MLMT) increased by 1.6 brightness levels compared to the control groupIn addition, in 1 year, 90% of patients with vision improvement of the brightness level of 1 brightness level, 65% of patients were able to achieve the minimum brightness level of 1 lux successfully through MLMT