The Food and Drug Administration said Tuesday [August 7] that Novartis submitted manipulated data as part of its application for a new gene therapy, and didn’t tell the agency until after the treatment was approved.
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said the inaccurate information involved mouse data and was a “small amount of the totality” that was submitted. The faulty data didn’t raise concerns about the safety and effectiveness of the treatment, he said, adding that the therapy will remain on the market.
The $2.1 million treatment — considered the world’s most expensive therapy — is called Zolgensma. It is used for children less than 2-years-old with spinal muscular atrophy, a leading genetic cause of infant death.
In a statement, Novartis said it is “fully confident in the safety, quality and efficacy of Zolgensma.” The drugmaker said AveXis investigated allegations of data manipulation and “once we had interim conclusions from our investigations, we shared our findings with the FDA.” The data, the company added, “are limited to an older process no longer in use.”
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