What data was manipulated in Zolgensma's application?
Animal-testing data used to demonstrate comparability between an early and final manufacturing process was manipulated — a small portion of the BLA not affecting human trial data, but disclosed to the FDA only on June 28, more than a month after the May 24 approval.
The manipulated data came from product testing in animals, used to show that an early version of Zolgensma was comparable to the later version made through a different manufacturing process. On August 6, 2019 the FDA disclosed that AveXis had informed it on June 28, more than a month after approval, about a data manipulation issue affecting the accuracy of certain data in the biologics licence application. The FDA said its concerns were limited to a small portion of the product testing data and did not change its positive view of the human clinical trials. The manipulated test was no longer in use and did not affect the approved product. The FDA kept Zolgensma on the market while it investigated.