Spark licenses blindness gene therapy rights outside US to Novartis

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

Philadelphia-based Spark said it will keep U.S. rights to Luxturna, or Vortigern 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.

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, which sells ophthalmology drugs including macular degeneration treatment Lucentis, last year won U.S. approval for Mariah, the first of a new type of potent gene-modifying immunotherapy for leukaemia.

“We think this deal is a positive for Spark, as the company will be able to focus on the U.S. launch of Luxturna, leaving the ex-U.S. launch and commercialization to Novartis, a leader in the ophthalmology space,” Jefferies analyst Michael Yee said in a note to investors.