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Why a drug that costs $2.1 million per dose is on a sales streak

January 29, 2020, 10:42 PM UTC

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Since its introduction in May, Novartis’s gene therapy medication, Zolgensma, has been the subject of plenty of controversy. For one thing, it’s the world’s most expensive drug by list price, coming in at a cool $2.1 million before discounts and rebates. And last August, a data manipulation scandal over the therapy, which was originally produced by AveXis, a biotech that Novartis snatched up in 2018, drew rebukes from the Food and Drug Administration (FDA)—though the therapy has remained on the market.

But the road bumps haven’t dampened sales of Zolgensma, the first-ever gene therapy approved to treat a devastating rare disease called spinal muscular atrophy. The muscle-wasting disorder typically affects children, and some forms of the disease usually lead to death before a child reaches his or her second birthday. And if the promise of treatment for the disease isn’t enough, the drug requires just a single dose, delivered via infusion, as a lasting cure for spinal muscular atrophy.

The hope for a forever cure appears to be driving Zolgensma’s success, high price and all. Novartis announced this week that the therapy brought in $186 million in sales in the fourth quarter of 2019 (and it’s only been on the market since late May). Major private insurers, as well as government programs like Medicaid, have been willing to cover the drug for most patients with the condition, according to Novartis.

This could well prove a critical test case in the gene therapy world. Drug manufacturers are betting that steep prices won’t matter (or, at least, won’t matter much) if they can prove that a therapy will prevent more drastic (and expensive) health conditions down the line, or save lives. Zolgensma’s early success may bolster that argument.

Zolgensma’s primary competitors are Biogen’s Spinraza and an experimental treatment from Roche called risdiplam, which is expected to be approved by May. Both of those treatments have significantly different formulations and delivery methods than Zolgensma.

Read on for the day’s news.

Sy Mukherjee


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