Spark Therapeutics acquires licence to supply viral gene therapy outside US

Spark Therapeutics has signed a licensing and supply agreement with Novartis Pharmaceuticals to develop and commercialise investigational gene therapy voretigene neparvovec outside the US.

The firm will retain its right for exclusive commercialisation of LUXTURNA (voretigene neparvovec-ryzl) in the US, and to obtain regulatory approval from the European Medicines Agency (EMA) for voretigene neparvovec.

LUXTURNA is an adeno-associated virus vector-based gene therapy indicated to treat patients suffering from confirmed biallelic RPE65 mutation-associated retinal dystrophy.

Under the agreement, Spark Therapeutics will receive an upfront payment of $105m and is eligible for additional $65m milestone-based payments based on the EMA approval and initial sales in select international markets.

"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."

The firm will also receive potential royalty payments on net sales of investigational voretigene neparvovec outside the US.

Spark Therapeutics chief business officer Dan Faga said: "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.

"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."

The firm additionally signed a separate agreement for the manufacture and supply of investigational voretigene neparvovec to Novartis.