Licensing and supply agreement for voretigene neparvovec outside the US

Spark Therapeutics announced it has entered into a licensing agreement with Novartis Pharmaceuticals to develop and commercialise investigational voretigene neparvovec outside the US, while Spark Therapeutics will continue to exclusively commercialise Luxturna (voretigene neparvovec-ryzl) in the US

Spark Therapeutics is a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease. Under the agreement, with Novartis Pharmaceuticals, Spark Therapeutics will retain regulatory responsibility for obtaining European Medicines Agency (EMA) approval for investigational voretigene neparvovec.

Spark Therapeutics also entered into a separate agreement to manufacture and supply investigational voretigene neparvovec to Novartis. No other programmes in Spark Therapeutics’ pipeline are part of this agreement.

Under the terms of the licensing agreement, Novartis will pay Spark Therapeutics $105 million in cash as an upfront fee.

Spark Therapeutics is eligible to receive up to an additional $65 million in cash milestone payments based on near-term EMA regulatory approval and initial sales outside the US in certain markets.

Spark Therapeutics is also entitled to receive royalty payments on net sales of investigational voretigene neparvovec outside the US.

“By leveraging Novartis’ large, existing commercial and medical infrastructure in ophthalmology, as well as its commitment to commercialising genetic-based medicines, we help ensure that more patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy who live outside the US and importantly outside of Europe, have access to investigational voretigene neparvovec,” said Dan Faga, Chief Business Officer, Spark Therapeutics.

“We intend to use the proceeds from this transaction to continue to develop our robust pipeline of investigational gene therapies to create a path to a world where no life is limited by genetic disease.”

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