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Spark Therapeutics' One-time Gene Therapy LUXTURNA Gets EU Approval

Spark Therapeutics (ONCE), a commercial gene therapy company, announced Friday that the European Commission has granted marketing authorization for LUXTURNA (voretigene neparvovec).

LUXTURNA is a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

This authorization is valid in all 28-member states of the EU, as well as Iceland, Liechtenstein and Norway. LUXTURNA is the first and only gene therapy approved in EU for patients with an inherited retinal disease or IRD.

It is also the first gene therapy for a genetic disease that has received regulatory approval in both the U.S. and EU.

RPE65-mediated IRD is a progressive condition leading to total blindness in most patients.

In January 2018, Spark entered into a licensing agreement with Novartis to commercialize LUXTURNA when approved in Europe and all other markets outside the U.S.

Spark will manufacture and supply LUXTURNA for Novartis, while Novartis has exclusive rights to pursue development, registration and commercialization in all other countries outside the U.S.

In September 2018, the Committee for Medicinal Products for Human Use or CHMP of the European Medicines Agency adopted a positive opinion recommending approval of LUXTURNA.

LUXTURNA was approved by the U.S. Food & Drug Administration (FDA) in December 2017.

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