Spark Therapeutics Inc
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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