Novartis' pricing of gene therapy products in spotlight as Luxturna gains EU clearance, Express Scripts weighs in on proposed cost of AVXS-101

Novartis announced Friday that the European Commission approved Luxturna as a one-time gene therapy for patients with vision loss due to a genetic mutation in both copies of the RPE65 gene and who have enough viable retinal cells. The clearance comes as US pharmacy benefits manager Express Scripts weighed in on the debate around proposed pricing of the gene therapy AVXS-101 for treating spinal muscular atrophy (SMA), which the Swiss drugmaker recently suggested could be cost-effective to healthcare systems at $4 million to $5 million per patient.

Steve Miller, Express Scripts chief medical officer, said although he "loves the science" behind AVXS-101, "you just can't keep pushing these price points up. I just don't think we can allow it. It is not sustainable over time." Novartis gained AVXS-101 earlier this year after buying AveXis for $8.7 billion, with FDA approval of the one-time gene therapy expected in early 2019.

Currently there is only one authorised treatment for SMA, Biogen and Ionis Pharmaceuticals' Spinraza (nusinersen), which needs to be taken on an ongoing basis at a cost of $750 000 for the first year and $350 000 annually thereafter. Dave Lennon, AveXis's president, commented "when we look at 10-year costs, you see somewhere between $2.5 million to $5 million being spent by societies to care for these types of patients," adding "$4 million is a significant amount of money, but we believe this is a cost-effective point."

The cost-effectiveness of SMA therapies, including AVXS-101, are currently being assessed in the US by the Institute for Clinical and Economic Review (ICER), with a final report due in March. The analysis is set to include a head-to-head comparison of Spinraza's and AVXS-101's financial impacts over time. However, gene therapies have not fared so well in ICER reviews, with the institute previously criticising the price of Luxturna in the US.

Luxturna, which was developed by Spark Therapeutics, was cleared by the FDA last year for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, with the company pricing the therapy at a cost of $850 000, or $425 000 per eye. However, ICER argued that Luxturna in most cases should cost about 75 percent less than the proposed price.

In Europe, Novartis indicated that "a range of resources and innovative reimbursement and access approaches are…being explored" for Luxturna. Paul Hudson, CEO of Novartis Pharmaceuticals, said that the gene therapy "can bring real value to patients, their families and society as a whole," adding that the company "is committed to working with patients, caregivers, health systems and physicians to establish access" to the product. According to Novartis, decisions from national reimbursement bodies in the EU on Luxturna are expected in 2019 and 2020.

Novartis gained rights to Luxturna outside the US from Spark in January in a deal worth potentially more than $170 million.

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