Leaders of AveXis Out Over Doctored Gene Therapy Data
Leaders of AveXis Out Over Doctored Gene Therapy Data

Leaders of AveXis Out Over Doctored Gene Therapy Data

Novartis, which bought the biotech firm in 2018, announced that the company’s top scientists have left, and news reports say it’s because they were involved in using manipulated data to get the gene therapy Zolgensma approved.

Kerry Grens
Aug 14, 2019


Brian and Allan Kaspar, brothers at the scientific helm of Novartis’s AveXis division, left the company in May, and sources tell both STAT and CNBC it’s because they were involved in an episode in which falsified data were submitted to the US Food and Drug Administration as part of an application for getting a gene therapy approved. 

Brain Kaspar was the chief scientific officer and Allan Kaspar was the head of research during the time when the company was working through the process of getting Zolgensma, a gene therapy for children with spinal muscular atrophy, approved.

The FDA only learned of the compromised preclinical data in June—a month after the agency gave the drug the green light, and a month after the Kaspar brothers left the company.

“The news will likely only amplify questions as to why Novartis delayed notifying the FDA of concerns,” STAT writes. 

Vas Narasimhan, the CEO of Novartis, tells CNBC that his team began investigating the situation once the problems came to light, but didn’t fill the FDA in until the company had “the best information and technical analysis, which we did promptly on completion on June 28.”

The fudged results concerned a potency test conducted in animals. The FDA says the gene therapy is still considered safe and effective for children, and it is investigating the situation—including the possibility of civil or criminal action.

Kerry Grens is a senior editor and the news director of The Scientist. Email her at kgrens@the-scientist.com.