Research & Development
Children's Hospital announces European Commission's first-of-its-kind gene therapy approval for blindness
6 December 2018 -

Paediatric hospital Children's Hospital of Philadelphia (CHOP) reported on Wednesday the receipt of approval from the European Commission (EC) for LUXTURNA (voretigene neparvovec) as the first and only gene therapy for patients with an inherited retinal disease.

This clearance makes LUXTURNA the first gene therapy for a genetic disease that has received regulatory approval in both the US and European Union (EU).

In December 2017, the US Food and Drug Administration (FDA) approved LUXTURNA for use in patients in the US. The gene therapy is currently administered at ten treatment centres by leading retinal surgeons who receive training provided by Spark Therapeutics on the administration procedure.

The EC approved the one-time gene therapy LUXTURNA for the treatment of vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations, in paediatric and adult patients who have sufficient viable retinal cells. RPE65 -mediated inherited retinal disease is a progressive condition that leads to total blindness in most patients. The EC authorization is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway.

Under a prior agreement, Spark Therapeutics had signed a licensing and supply agreement with Novartis covering development, registration and commercialisation rights to LUXTURNA in markets outside the US. Novartis can commercialise LUXTURNA in the EU/EEA. Novartis already has exclusive rights to pursue development, registration and commercialisation in all other countries outside the US and Spark Therapeutics will supply the gene therapy to Novartis.



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