EU panel recommends clearance of Novartis' gene therapy Luxturna for inherited retinal disease

Novartis said Friday that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion regarding approval of Luxturna (voretigene neparvovec) for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. The one-time gene therapy was developed and is marketed in the US by Spark Therapeutics. 

The CHMP recommendation was backed by safety and efficacy data from three clinical studies involving a total of 43 patients with inherited retinal disease due to mutations in both copies of the RPE65 gene. In Phase III study data published in The Lancet, Luxturna was associated with significant improvement in functional vision, as measured by the binocular multi-luminance mobility test (MLMT) score change from baseline to one year, versus the control.

Novartis gained rights to Luxturna outside the US from Spark Therapeutics in January in a deal worth potentially more than $170 million. Paul Hudson, CEO of Novartis Pharmaceuticals, said "we look forward to working with Spark Therapeutics and the European Medicines Agency to establish access and reimagine care for people in the EU who face the threat of total blindness from this inherited retinal disease." 

A final decision from the European Commission regarding approval of Luxturna is expected within two months. The gene therapy was submitted for European approval in July 2017. 

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Luxturna was cleared by the FDA last December for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Spark later priced the therapy at a cost of $850 000, or $425 000 per eye, drawing criticism from the Institute for Clinical and Economic Review, which argued that Luxturna in most cases should cost about 75 percent less than the proposed price. The treatment generated $6.7 million in US revenue in the first half, but some analysts predict sales will climb to more than $300 million by 2022.

Beyond its partnership with Spark for Luxturna, Novartis' expansion into gene and cell therapy also includes the CAR-T cell therapy Kymriah (tisagenlecleucel), which is approved for advanced blood cancer in Europe and the US, while the prospective gene therapy AVXS-101 for spinal muscular atrophy came with the completion of its $8.7-billion takeover of AveXis earlier this year.

Novartis CEO Vas Narasimhan remarked "we have three, but of course we're also looking for more opportunities, with a focus on certain therapy areas where we already have a presence…That's something we're actively looking at."

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