FDA Looks Into Data Manipulation, Poor Record-keeping at Novartis Subsidiary

The FDA is investigating a Novartis subsidiary over “data manipulation” reported only after the agency approved its gene therapy Zolgensma (onasemnogene abeparvovec-xioi).

The issue came to light when AveXis reported problems with animal testing data submitted in the BLA for the gene therapy product for treating spinal muscular atrophy. The product was approved on May 24, 2019. AveXis alerted the agency to the problem on June 28.

CBER Director Peter Marks said Tuesday that the agency “will use its full authorities to take action, if appropriate,” and civil or criminal penalties could follow. Despite its concerns, the agency “remains confident” that the product should remain on the market, he said.