How this gene therapy drug earned its $2.1 million price tag

Fortune

8 February 2020 - Swiss drugmaker Novartis recently launched a lottery-style program to allocate free doses of Zolgensma, its groundbreaking treatment for children with spinal muscular atrophy, to patients in countries that haven't approved the drug.

But with a $2.1 million price tag, Zolgensma is inaccessible to some patients even where it has been approved.

The prohibitive cost has been a source of criticism since the drug was approved. In a letter to the U.S. Food and Drug Administration's (FDA) Acting Commissioner last August, five U.S. senators, including presidential hopefuls Elizabeth Warren and Bernie Sanders, accused AveXis, the subsidiary of Novartis responsible for the drug, of falsifying testing data to gain FDA approval in a move that “smacks of the pharmaceuticals industry's privilege and greed." Meanwhile, the Boston-based Institute for Clinical and Economic Review, a research group that evaluates drug pricing, claims Novartis could reasonably price Zolgensma in the $310,000 to $900,000 range.

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Michael Wonder

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Michael Wonder

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US , Pricing , Gene therapy