Novartis scores EU nod for gene therapy Luxturna

It’s been almost a year since Spark Therapeutics won a groundbreaking gene therapy approval in the U.S. for Luxturna, and now its marketing partner Novartis has done the same across the pond.

The one-time drug secured EU approval to treat patients suffering from vision loss due to a mutation in the RPE65 gene, Novartis said. The drugmaker is now working on "innovative" payment plans and expects to finalize reimbursement deals in individual markets in 2019 and 2020.

Luxturna treats a rare genetic mutation that causes vision loss at an early age and usually progresses to blindness. Spark Therapeutics developed the drug and secured FDA approval last December. In the U.S., Spark priced Luxturna at $850,000, or $425,000 per eye before discounts.

In a statement, Novartis’ pharma CEO Paul Hudson said the EU approval "marks a milestone in reimagining medicine and can bring real value to patients, their families and society as a whole." 

Novartis is "committed to working with patients, caregivers, health systems and physicians to establish access to this gene therapy for RPE65 patients, as we believe it can help restore sight and improve vision in children and adults who currently have no treatment options," Hudson added.

With the approval, Novartis said it’s looking at a "range of resources and innovative reimbursement and access approaches" to help fund the drug. The company, which won the world’s first CAR-T approval for Kymriah in 2017, picked up ex-U.S. rights to Luxturna in a January deal worth up to $170 million.

In its U.S. launch, Spark has worked to establish outcomes-based payment deals and ways for payers to fund the drug over several years. 

Meanwhile, after an April buyout of AveXis worth $8.7 billion, Novartis is working on AVXS-101 as a potential cure for spinal muscular atrophy. On a recent investor call, AveXis president Dave Lennon said the gene therapy could be cost effective at a price of $4 million to $5 million, instantly setting off conversation about that figure. Now, Express Scripts’ chief medical officer Steve Miller has weighed in.

In a Reuters interview, Miller said such prices are "not sustainable over time." 

"You just can’t keep pushing these price points up," Miller told the news service. 

The price tag would be for a drug to cure the deadly disease rather than treat it. Currently, Biogen’s Spinraza treats SMA at a price of $750,000 for the first year and $375,000 for subsequent years, before discounts. 

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