What happened with the Zolgensma data manipulation?
AveXis knew of manipulated animal-testing data before the FDA approved Zolgensma in May 2019 — but waited until June 28 to tell regulators, more than a month after approval. The FDA said it would likely have delayed the process had it known.
Zolgensma is a one-time gene therapy for spinal muscular atrophy, approved in May 2019 at a list price of $2.125 million. On August 6, 2019, the FDA revealed that Novartis's AveXis unit had told it on June 28, after approval, about manipulated data from animal testing submitted in the application, and that AveXis had known about the problem before approval.
The FDA said the drug should stay on the market because the issue did not affect human trial data, but warned that it could pursue civil or criminal penalties. The agency later said it would likely have delayed approval had it known. Novartis said it waited to complete an internal investigation before informing regulators and removed the scientists involved.