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CAMBRIDGE, Mass. — Days before his company is expected to win approval of the most expensive drug ever, the head of Swiss drug giant Novartis said Wednesday he could justify charging up to $5 million for the revolutionary treatment for a rare genetic disease, but it will end up being priced at a fraction of that.

Speaking at the company’s biomedical research institute, Novartis CEO Vas Narasimhan wouldn’t disclose the price of the drug, called Zolgensma, until the government approves it, probably by the end of the month. Zolgensma treats spinal muscular atrophy, or SMA, a deadly neurodegenerative disease.


Nonetheless, he said the one-time gene therapy treatment is so effective in infants that Novartis could charge $5 million and it would still be more economical than the current standard of care for spinal muscular atrophy.

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  • How can the cost of SMA1 be calculated at “~ $2M p.a over 10 years”, when NONE of these patients live to even 5 years.
    Cost-effectiveness is truly important, but the calculations quoted make utter nonsense of reality.
    STAT – surely you can do better?

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