FDA: AveXis manipulated potency data for Zolgensma

FDA said Tuesday that AveXis manipulated in vivo potency data for gene therapy Zolgensma and failed to report the issue to the agency, holding back the information while the agency was reviewing its BLA. The company’s alleged decision to stay mum on the issue at first could open it up for criminal and/or civil penalties, according to CBER Director Peter Marks.

The agency does not plan to pull the spinal muscular atrophy (SMA) therapy from the market and said it remains confident in its benefit-risk profile...