Novartis on Wednesday said it has made a with deal Spark Therapeutics about the rights for voretigene neparvovec in markets outside the US.
Voretigene neparvovec, known as Luxtarna in the US, was approved by the FDA in December last year, as a one-time gene therapy to reset functional vision in 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.
Novartis stressed out that there is currently no existing therapy for this disease outside the US.
“No otherwise healthy child should have to go blind due to this devastating disease. Gene therapy is a promising new avenue to potentially address this unmet need,” said Shreeram Aradhye, Global Head of Medical Affairs and Chief Medical Officer, Novartis Pharmaceuticals.
Novartis will make an upfront payment as well as pay milestones and royalties to Spark Therapeutics reflective of the late stage of the opportunity. Spark Therapeutics retains exclusive rights for LUXTURNA(TM) in the US and will retain responsibility for obtaining EMA approval. Commercialization rights will be transferred to Novartis upon successful completion of registration and issuance of market authorization. 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.