Spark licenses blindness gene therapy rights outside U.S. to Novartis

Spark Therapeutics Inc , whose breakthrough gene therapy to treat a rare form of blindness was approved by U.S. regulators in December, has licensed rights to the drug in markets outside the United States to Switzerland-based Novartis AG , it said on Wednesday.

Philadelphia-based Spark said it will keep U.S. rights to Luxturna, or voretigene neparvovec, which is the first approved gene therapy for an inherited disease. It plans to launch the one-time treatment in March, charging an unprecedented $850,000.

Spark will receive $105 million from Novartis and up to $65 million in milestone payments based on near-term European regulatory approval and initial sales in certain markets. Spark is also entitled to future royalties on net sales outside the United States.

In a statement, Spark said the deal leverages Novartis` "large, existing commercial and medical infrastructure in ophthalmology, as well as its commitment to commercializing genetic-based medicines."

Novartis last year won U.S. approval for Kymriah, the first of a new type of potent gene-modifying immunotherapy for leukemia.

Luxturna treats inherited retinal disease caused by defects in a gene known as RPE65, which affects between 1,000 and 2,000 people in the United States. It works by delivering by an eye injection viral vector particles containing a correct copy of the gene to retinal cells, restoring their ability to make a needed enzyme.

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