The price tag creates 'sticker shock.' But Novartis says that because the disease is expensive to treat, the drug is worth it in the long run.
as early as half a year before Friday's FDA approval, and it started negotiations with health insurers before the approval, too.
An estimated 10,000 to 25,000 individuals in the US live with the condition, according to the SMA Foundation. Only babies who are under two years old would likely be eligible for Zolgensma, Novartis said on Friday, and the company expects about 1,100 people to be eligible for it at launch.The severe nature of this rare disease is also why Novartis and AveXis believe that $2.1 million is actually a cost-effective price for the US healthcare system.
Spinal muscular atrophy is already very expensive to treat, and Zolgensma is expected to have long-term benefits for those who get it, Lennon and other Novartis executives said this week. Biogen's Spinraza, for instance, was the first-ever drug approved for spinal muscular atrophy and costs up to $750,000 for the first year of treatment and $375,000 in subsequent years. Like most medications, Spinraza is not a one-time treatment and must be taken consistently by patients.Friday press release"We have to remember this is a new therapy which has unique constructs that made us think differently about how we wanted to approach this," Lennon told Business Insider.
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