Novartis Licenses Spark’s Retinal Gene Therapy Outside US in $170 Million Deal

January 26, 2018: By Jon Swedien

Voretigene neparvovec

Novartis will develop and commercialize Spark Therapeutics’ gene therapy, voretigene neparvovec, outside the US under a licensing deal potentially worth more than $170 million, Spark announced Jan. 24.

Philadelphia-based Spark will continue to commercialize the gene therapy in the US under the brand name Luxturna.

Novartis, of Basel, Switzerland, will pay Spark $105 million in cash up front, and Spark is eligible to receive up to an additional $65 million in cash milestone payments and a royalty on net sales outside the US, Spark said.

The additional payments will be based on near-term European regulatory approval and initial sales outside the US in certain markets, the companies said.

Spark will retain regulatory responsibility for obtaining European Medicines Agency approval for the gene therapy. Spark also entered into a separate agreement to manufacture and supply investigational voretigene neparvovec to Novartis, the companies said.

On Dec. 19, the US FDA approved Luxturna for the treatment of retinal dystrophies caused by mutations in the RPE65 gene. Those dystrophies include a form of Leber congenital amaurosis (LCA2), a rare form of retinitis pigmentosa (RP 20), and severe early childhood onset retinal dystrophy (SECORD). LCA2 is the most common of them.

Luxturna is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease, and the first adeno-associated virus (AAV) vector gene therapy approved in the US, according to Spark.

Dan Faga, Spark’s chief business officer, said the company intends to use the proceeds to continue to develop its pipeline of investigational gene therapies.

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