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Novartis gets EU nod for Luxturna gene therapy

Approval comes almost a year after it was secured a green light in the US


Novartis has secured EMA approval Luxturna, a gene therapy for a rare eye disease, almost a year after it was given the green light in the US.

Luxturna (voretigene neparvovec) has been cleared for the treatment of adults and children suffering from inherited retinal dystrophy (IRD) caused by RPE65 gene mutations, a rare genetic disorder which causes vision loss and usually leads to blindness. Patients have to have sufficient viable retinal cells remaining in order for the treatment to work.

Novartis secured rights to the gene therapy outside the US from its developer Spark Therapeutics in January, paying $100m upfront, shortly after Spark won FDA approval for the therapy as the first  directly-administered gene therapy in the US.

Spark reported almost $9m in the third quarter from the sale of 24 vials of the therapy, twice the number shipped in the prior three-month period which was in turn twice the number shipped in the first, which the company said was a strong performance.

That works out at around $375,000 per vial, which is enough to treat one eye, around $50,000 less than the list price at launch, although the increasingly influential Institute for Clinical and Economic Review (ICER) has suggested the price of Luxturna should come down 75% from list to make it cost-effective.

Spark has offered a rebate scheme tied to clinical outcomes, however, which will pay back around a quarter of the price in the form of a rebate if visual improvement objectives are not reached after 30 months. Novartis hasn’t yet indicated its pricing plans for Luxturna in Europe or made any comment on possible rebate systems, but has said it will look at ways to ensure patients are not denied therapy on cost grounds.

Paul Hudson, chief executive of Novartis’ pharma business, said in a statement that the company “is committed to working with patients, caregivers, health systems and physicians to establish access” to the therapy” and that decisions from national reimbursement bodies – including NICE and the Scottish Medicines Consortium – “are expected in 2019 and 2020.”

This type of IRD affects somewhere between 1,000 and 2,000 people in the US with around 10 new cases per year, and a similar number in the EU.

The approval of Luxturna came as US pharmacy benefits manager Express Scripts weighed into suggestions by Novartis during its third-quarter results call that one of its experimental gene therapies – AVXS-101 for spinal muscular atrophy – could still be cost-effective if priced at $4m or more.

Express Scripts’ chief medical officer, Steve Miller, told Reuters that regardless of the potential of a new product “we can’t keep pushing these price points up. It’s not sustainable over time”.

Article by
Phil Taylor

26th November 2018

From: Regulatory



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