The Food and Drug Administration is threatening to take criminal action against Novartis, saying that the drugmaker used data it knew was incorrect in its application for a $2.1 million gene therapy that was approved in May. The FDA said the handled data involved testing in animals, not patients, and it’s confident that the drug, called Zolgensma, should remain on the market. The FDA said it’s investigating and will consider criminal or civil penalties.
Zolgensma was approved for children under 2, becoming the most expensive treatment ever. It’s a treatment for a rare inherited condition, spinal muscular atrophy, which destroys a baby’s muscle control. The agency said the Novartis knew about the issue before the FDA approved Zolgensma.
The inaccurate data is a small subgroup of the testing information that the FDA estimated. Novartis said that after AveXis learned of alleged data manipulation in one animal testing procedure, the company straightaway began investigating
Novartis didn’t say why it didn’t inform the FDA before it approved Zolgensma. Novartis specified that the animal test in question isn’t used in making the therapy for patients and that Zolgensma is safe and effective. FDA inspected the company’s Irvine, California, manufacturing plant in recent weeks, and then issued a report finding several deficiencies, including not fully following quality control procedures.