Swiss drugmaker Novartis has shaken up the management at its gene therapy subsidiary in the wake of the data manipulation scandal that rocked the company last week.
The subsidiary, AveXis, said Tuesday it had appointed Page Bouchard as Senior Vice President of research and chief scientific officer, effective Aug. 5. The announcement comes the week after the Food and Drug Administration stated publicly that some preclinical data in the submission package of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) – approved in May for spinal muscular atrophy in infants – had been manipulated.
Bouchard replaces Brian and Allan Kaspar, brothers who had respectively served as head of research and development and chief scientific officer, roles that will be combined into Bouchard’s position. The company said the Kaspars “have not been involved in any operations at AveXis since early May 2019 and are no longer with the company.”
Citing an unnamed source, STAT reported Wednesday that the Kaspars were ousted from the company in connection with the data manipulation scandal.
In response to a request for comment, a Novartis spokesperson referred to the AveXis announcement and reiterated the timeline of events connected to the data manipulation that the company had provided, but declined to comment further.
The FDA had said last week that the Zolgensma application included manipulated mouse data, which Novartis said came from an assay that is no longer used. While the agency said the data manipulation did not raise concerns about benefit-risk profile of the gene therapy, which the agency approved in May for spinal muscular atrophy in infants, AveXis was apparently aware of it as early as March, but did not tell the agency until June. Novartis has said it stands by the therapy and that the delay in disclosing the manipulation was so that it could complete an internal investigation, not for expediency of approval, which the FDA said would have been delayed had the company disclosed the findings ahead of Zolgensma’s approval.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
The scandal has since led to political fallout. Last week, five Democratic senators – including presidential candidates Bernie Sanders of Vermont and Elizabeth Warren of Massachusetts – sent a letter to FDA acting Commissioner Ned Sharpless urging the agency to take action. Also last week, Sen. Chuck Grassley, R-Iowa, sent a letter to Novartis CEO Vas Narasimhan, calling the allegedly intentional withholding of data “reprehensible” and asking for information on, among other things, when the company learned of the data manipulation and requesting records pertaining to the incident and the investigation.
And on Tuesday, The New York Times’ editorial board urged lawmakers and regulators to limit the use of accelerated approval, cap prices for medications whose risk-benefit profiles are not yet proven and demand more data to prove them.
Photo: Novartis
