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Novartis delayed notifying about gene therapy data manipulation until after approval, FDA says

BMJ 2019; 366 doi: (Published 09 August 2019) Cite this as: BMJ 2019;366:l5109
  1. Owen Dyer
  1. Montreal

The US Food and Drug Administration has strongly criticised the Novartis subsidiary AveXis for failing to immediately pass on its discovery that data had been manipulated in the early stages of testing Zolgensma (onasemnogene abeparvovec-xioi), a new gene therapy for spinal muscular atrophy, which at $2.1m (£1.7m; €1.9m) a dose is the most expensive drug in the world.

“AveXis became aware of the issue of the data manipulation that created inaccuracies in their biologics licence application before the FDA approved the product, yet did not inform the FDA until after the product was approved,” said Peter Marks, director of the FDA’s biologics division, …

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