In an inspection of the Illinois manufacturing facility for Novartis’ Zolgensma earlier this year, a team of top FDA officials met with AveXis staff. Although the assay that is now at the center of Novartis scandal was discussed, the FDA found the whole situation “unremarkable.”
That is because the AveXis team, which included the now-dispatched Allan Kaspar, explained away errors. They told the FDA that there had been some issues with the original assay for determining the potency of the gene therapy, but that they had been corrected. Poor reporting was the culprit, they said, and a new procedure was put in place with “new forms for documentation and to clarify and standardize.”
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The FDA inspector, Burnell Henry, said he reviewed the new procedure and the document which “corrects multiple errors in the report,” including adding results from a mouse test subject that had been “inadvertently omitted in the first version of the report,” according to a 14-page, highly-redacted Establishment Inspection Report.
The FDA had some suggestions for AveXis. The new procedure needed to be added to the company’s drug application, and better record keeping was needed, but, all in all, the FDA found the situation unremarkable.
It was a month later that an AveXis scientist stepped forward to tell Novartis that some of the data included in the application were inaccurate. Two months later, after the FDA had already approved Zolgensma, Novartis broke the news of the faked data to the FDA.
The whole episode has blown up for the Swiss drugmaker. The FDA has said that the drug is still safe and effective and will remain in circulation, but the agency is investigating what happened during that two-month delay. It has threatened civil or criminal penalties.
Novartis CEO Vas Narasimhan has acknowledged the company could have handled the situation better but insists the drugmaker didn’t mislead the FDA. He said it just didn’t want to inform the agency until it knew exactly what had happened and whether the falsification put patients at risk.
The company has fired a small group of scientists who knew or should have known about the manipulation. That includes Allan Kaspar, who was AveXis’ senior vice president of R&D, and his brother Brian, who was chief medical officer. Brian Kaspar insists he has done nothing wrong and hired a high-powered attorney to defend his position.
The FDA investigation continues. In addition, Sen. Chuck Grassley, chair of the Senate Finance Committee, has launched his own and requested all records related to the incident.
Also, a group of senators has demanded an explanation from the agency as to why last year it scrapped a proposal that would have required trial sponsors to report information suggesting any person “has, or may have, engaged in the falsification of data.” The rule was killed by the FDA last October, the same month AveXis was supposedly fixing its data problems.