ICER suggests Novartis' gene therapy Zolgensma could be more cost-effective than Biogen, Ionis' Spinraza for spinal muscular atrophy

According to a draft evidence report released by the Institute for Clinical and Economic Review (ICER), Biogen and Ionis Pharmaceuticals' Spinraza (nusinersen) treatment for spinal muscular atrophy (SMA), as well as Novartis' proposed SMA gene therapy Zolgensma (onasemnogene abeparvovec), are both expensive, but the gene therapy could be more cost-effective once additional information is known about its US price and long-term success rates. 

In its preliminary assessment, ICER concluded that Spinraza "appears to be most cost-effective when used in patients with presymptomatic SMA," at a cost of $728 000 per quality-adjusted life year (QALY), which ICER chief medical officer David Rind stated "is expensive by the way most people value things." The drug, which was cleared in 2016 and is the only FDA-approved treatment for SMA in the US, costs $750 000 for the first year and $375 000 for each subsequent year of therapy. 

Meanwhile, ICER said that when used in patients with symptomatic type I SMA, Zolgensma would represent a cost of $240 000 per QALY, although the group cautioned that the figure was based on an estimated list price of $2 million for the gene therapy. The FDA is assessing Zolgensma under a priority review, with approval expected in the first half of next year. Novartis, which gained rights to the therapy, also known as AVXS-101, via its $8.7-billion purchase of AveXis earlier this year, has said the final price will be set after discussions with insurers, but recently indicated that it could be cost-effective at a price of $4 million to $5 million per patient as a one-time treatment.

Novartis spokesman Eric Althoff expressed that the drugmaker wants Zolgensma to be compared against a threshold of $500 000 per QALY and against Spinraza. "While a range of QALY thresholds were used, we believe cost-effectiveness assessments in SMA should be compared to currently available, chronic therapy and be assessed at the ultra-rare disease QALY threshold," Althoff stated. 

ICER plans to issue a final assessment of the cost-effectiveness of Spinraza and Zolgensma in February. Last month, Express Scripts questioned whether Novartis' projected price of Zolgensma would be sustainable over time. 

Meanwhile, third-quarter sales of Spinraza surged 73 percent year-over-year to $468 million, exceeding forecasts of $448 million. 

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