A gene therapy that was the first of its kind to receive Food and Drug Approval in the US has won approval from Europe’s main drug regulator as well.

Swiss drugmaker Novartis said Friday that the European Commission approved Luxturna (voretigene neparvovec-rzyl) for patients with vision loss due to a genetic mutation of the RPE65 gene. The approval covers the 28 nations of the European Union – including outgoing member the United Kingdom – and the three countries in the European Economic Area, namely Norway, Iceland and Liechtenstein. Luxturna’s original developer, Philadelphia-based Spark Therapeutics, markets the therapy in the US.

Luxturna is a viral vector-based gene therapy that uses an adeno-associated viral vector to provide a working copy of the RPE65 gene to act in place of the mutated gene that leads to vision loss in patients with RPE65-associated inherited retinal disease, or RPE65-associated IRD. The disease is estimated to affect 1-in-200,000 people, according to Novartis, while Spark estimates the total population in the US, Europe, Asia-Pacific and elsewhere in the Americas is up to 6,000 people.

The list price of the therapy is $850,000 – $425,000 per eye – in the US, where the FDA approved it in December 2017. The following month, Spark announced programs devised with Harvard Pilgrim and affiliates of pharmacy benefit manager Express Scripts as well as the Centers for Medicare and Medicaid Services to provide coverage for the therapy. These include outcomes-based rebates with long-term durability measures and a contracting model designed to support access to Luxturna.

In Europe, Novartis will have to go to individual states’ authorities that handle drug pricing and reimbursement negotiations and said it expects decisions next year and in 2020. These include the UK’s National Institute for Health and Care Excellence, or NICE, which typically mandates that manufacturers offer discounts and rebates sufficient to bring the cost per quality-adjusted life year, or cost per QALY – officially known as the incremental cost-effectiveness ratio, or ICER – into a range of 20,000-30,000 pounds, or $25,496-$38,244. That is the threshold NICE requires in order to recommend a therapy as cost-effective for routine use by the National Health Service.

Two weeks ago, NICE recommended that another high-priced and logistically complex Novartis therapy, the CAR-T cell therapy Kymriah (tisagenlecleucel), be made available for children and young adults with acute lymphoblastic leukemia. But Kymriah’s US list price is much less than Luxturna’s – $475,000 – while the NICE-quoted list price for the UK was equivalent to less than $362,000. Moreover, NICE recommended Kymriah not for routine use by the NHS, but for coverage through the Cancer Drugs Fund – which only covers cancer therapies, not treatments for orphan genetic disorders like the one Luxturna treats – pending collection of more data to resolve ongoing uncertainties around its cost, even though the pricing watchdog acknowledged Kymriah is a “highly innovative treatment.”

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