Novartis proclaimed a licensing agreement with Spark Therapeutics encompassing development, registration and commercialization rights to voretigene neparvovec in markets outside the US. Voretigene neparvovec, known as LUXTURNA(TM) (voretigene neparvovec-rzyl) in the US that received FDA approval on December 19, 2017 as a one-time gene therapy to restore functional vision in children and adult patients with biallelic mutations of the RPE65 (retinal pigment epithelial 65 kDa protein) gene. The market authorization application (MAA) with the European Medicines Agency (EMA) was filed on July 31, 2017. The US is the only country to claim existing therapy. Novartis has exclusive rights to pursue development, registration and commercialization in all other countries outside the US. Spark Therapeutics will be responsible for the supply of voretigene neparvovec worldwide under a separate manufacturing and supply agreement with Novartis.
Shreeram Aradhye, Global Head of Medical Affairs and Chief Medical Officer, Novartis Pharmaceuticals said “This collaboration builds on our commitment to ophthalmology. We look forward to leveraging our global presence to ensure that patients outside the US have access to this potentially life-changing treatment.”
Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle and resulting in progressive vision loss and ultimately, blindness. Only a few thousand people worldwide are affected by this ultra-orphan condition. Pending approval, Novartis will work with physicians to embark on revolutionizing fresh approaches to diagnose and treat specialized treatment centers.