On May 24, the FDA approved Zolgensma, a gene therapy product used to treat children less than two years of age with spinal muscular atrophy (SMA). On June 28, the FDA was informed by AveXis Inc. about a data manipulation issue that impacts the accuracy of data from animal testing submitted in the biologics license application (BLA). According to the agency “We are carefully assessing the issue of the manipulation of the product testing data used in the production process and are conducting a thorough assessment of the information from a recently completed inspection.” The manipulated data came from an animal test comparing two versions of Zolgensma, one used in a Phase 1 study and another tested in Phase 3.
The agency also stated: “We are also aware that AveXis became aware of the issue of the data manipulation that created inaccuracies in their BLA before the FDA approved the product, yet did not inform the FDA until after the product was approved. The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties.”
Wilson Bryan, head of the FDA's Office of Tissues and Advanced Therapies, indicated in an agency memo that had the FDA known of the data issues, it wouldn't have approved Zolgensma when it did.
Brian and Allan Kaspar were the chief scientific officer and vice president at AveXis, respectively. Novartis purchased AveXis for $8.7 billion in 2018. Brian Kaspar made over $380 million off of his stock holdings in the company through the deal.
According to a company statement on August 14, “Brian Kaspar and Allan Kaspar have not been involved in any operations at AveXis since early May 2019 and are no longer with the company.” In the statement, the company announced that Page Bouchard, a DVM with 27 years of industry experience, will take over both roles previously held by the Kaspars.
Why is this important?
If you read our article about the Wellcome Global Monitor study, and you have heard about the recent release of the Pew Research report on "Trust and Mistrust in American Views of Scientific Experts” then you know that public trust is at a seemingly all time-high. But scandals like the Novartis Zolgensma controversy threaten to break that trust and move people towards more skeptical opinions.
Currently, public confidence in scientists is on par with the military, according to the Pew report. But Americans are divided in terms of how they view the value and objectivity of scientists and their ability to act in the public interest. Incidents, including the Novartis Zolgensma case, can be viewed as scientists acting for personal gain. The disregard for public safety may place additional doubt in the public minds, especially when they don’t not fully understand the scientific basis or regulatory processes.
Moreover, scientific integrity of companies and scientists can be called into question. Between a quarter and half of Americans consider misconduct a “very big” or “moderately big” problem. Explosive news stories resulting from the Novartis controversy may exacerbate these concerns among the public. This controversy and other similar cases over time may explain why the public believe that scientists lack transparency (81% or greater).
While the FDA in its Wednesday statement assured the public that Zolgensma remains perfectly safe for use and Novartis stands behind its product. The doubt cast upon the company may do unescapable damage. This incident provides us of why scientists, in any field, have a mission that is just as important as the search for innovation and that is to work in and for the public interest.
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