AveXis top scientists out after data manipulation surfaces 

Swiss biologics firm AveXis Inc., which recently took over AstraZeneca’s former pharmaceutical production facility in Longmont, has fired two of its top scientists in the wake of data manipulation related to the company’s U.S. Food and Drug Administration application for gene therapy Zolgensma.

Brothers Brian Kaspar and Allen Kaspar respectively AveXis’ chief scientific officer and senior vice president of research and development — are no longer employed by the firm and “not been involved in any operations at AveXis since early May 2019,” according to an AveXis announcement. The company does not specifically cite the data manipulation accusations as a reason for the Kaspar brothers’ termination.

Page Bouchard, global head of preclinical safety for Novartis Institutes for BioMedical Research, has been tapped to take over the duties of both CSO and SVP of research and development. AveXis is a Novartis affiliate. 

The data manipulations were announced earlier this month by the FDA.

“On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children younger than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene — the most severe form of SMA. SMA is a leading genetic cause of infant mortality,” according to the FDA’s announcement. “Subsequently, on June 28, following the FDA’s approval of the product, the agency was informed by AveXis Inc., the product’s manufacturer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA.”

Because “AveXis became aware of the issue of the data manipulation that created inaccuracies in its BLA before the FDA approved the product, yet did not inform the FDA until after the product was approved,” regulators may pursue civil or criminal penalties, according to the FDA.

“We made the decision to progress our quality investigation prior to informing FDA and other regulatory authorities so that we could provide the best information and technical analysis, which we did promptly on completion on June 28,” Novartis CEO Vasant Narasimhan told investors and analysts during a conference call earlier this month.

Despite the data manipulation, the FDA is standing by its decision to approve Zolgensma.

“The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application,” according to a statement from the regulatory body. “This product testing data was used by the manufacturer to support the development of its production process for the product. These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program.”

AveXis and Novartis are also still in support of the drug.

“We maintain that the totality of the evidence demonstrating the product’s effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile,” according to a statement from AveXis.

Swiss biologics firm AveXis Inc., which recently took over AstraZeneca’s former pharmaceutical production facility in Longmont, has fired two of its top scientists in the wake of data manipulation related to the company’s U.S. Food and Drug Administration application for gene therapy Zolgensma.

Brothers Brian Kaspar and Allen Kaspar respectively AveXis’ chief scientific officer and senior vice president of research and development — are no longer employed by the firm and “not been involved in any operations at AveXis since early May 2019,” according to an AveXis announcement. The company does not specifically cite the data manipulation accusations as a reason for the Kaspar brothers’ termination.

Page Bouchard, global head of preclinical safety for Novartis Institutes for BioMedical Research, has been tapped to take over the duties of both CSO and SVP of research and development. AveXis is a Novartis affiliate. 

The data manipulations were announced earlier this month by the FDA.

“On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children younger than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene — the most severe form of SMA. SMA is a leading genetic cause of infant mortality,” according to the FDA’s announcement. “Subsequently, on June 28, following the FDA’s approval of the product, the agency was informed by AveXis Inc., the product’s manufacturer, about a data…