Industry News
One-time Gene Therapy for Inherited Retinal Disease Approved By European Commission
Spark Therapeutics, a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, recently announced that the European Commission has granted marketing authorization for LUXTURNA® (voretigene neparvovec), 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. RPE65-mediated inherited retinal disease (IRD) is a progressive condition leading to total blindness in most patients. This authorization is valid in all 28-member states of the EU, as well as Iceland, Liechtenstein and Norway. LUXTURNA is the first gene therapy for a genetic disease that has received regulatory approval in both the U.S. and EU.
“The historic approval of LUXTURNA in Europe furthers our mission to challenge the inevitability of genetic disease around the world. Following the launch of LUXTURNA in the United States earlier this year, this decision makes LUXTURNA the first gene therapy for a genetic disease approved in both the U.S. and EU, a promising milestone for the many people living with genetic disease around the world,” said Ron Philip, chief commercial officer at Spark Therapeutics. “Having worked closely with the global IRD community on the development of LUXTURNA, we are proud to be able to share the first gene therapy treatment option for an inherited retinal disease (IRD) with appropriate patients in Europe, in collaboration with Novartis.”
In January 2018, Spark Therapeutics entered into a licensing agreement with Novartis to commercialize LUXTURNA when approved in Europeand all other markets outside the U.S. Under the licensing agreement, Novartis has exclusive rights to pursue development, registration and commercialization in all countries outside the U.S. Upon the transfer of the marketing authorization from Spark Therapeutics to Novartis, Novartiscan commercialize LUXTURNA in the EU/European Economic Area (EEA). Spark Therapeutics entered into a separate agreement with Novartis to manufacture and supply LUXTURNA to Novartis.
“Today’s approval is momentous for patients given that there have been no pharmacological treatment options to date,” said Christina Fasser, president of Retina International, an umbrella organization of more than 43 patient organizations worldwide promoting research to find treatments for inherited retinal degenerative diseases. “Access to this treatment has the potential to reduce the substantial physical, emotional and financial burden this disease has on patients and their families.”
In September 2018, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of LUXTURNA. This decision was 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 in both copies of the RPE65 gene. The Phase 3 trial was the first randomized, controlled gene therapy trial for a genetic disease. LUXTURNA is designated as an orphan medicinal product for the treatment of inherited retinal dystrophies.
LUXTURNA was approved by the U.S. Food & Drug Administration (FDA) in December 2017.
