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Novartis Addresses FDA Data Manipulation Inquiry

Posted on August 07, 2019 by Medtech[y] Staff

The FDA announced they were informed by AveXis, Inc., the gene therapy subsidiary of Novartis, that data was knowingly manipulated and inaccurate in their application for Zolgensma, a $2.1 million gene therapy which was approved on May 24. While the FDA was alerted, it wasn't until after the therapy was approved.

"On June 28, following the FDA’s approval of the product, the agency was informed by AveXis Inc., the product’s manufacturer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA," said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research.

The agency is threatening to take criminal or civil action against Novartis.

While the FDA believes Novartis knew the data was inaccurate, the agency still believes the gene therapy product intended to treat children less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene, should remain on the market during the investigation.

"The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application. This product testing data was used by the manufacturer to support the development of its production process for the product. These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program. The totality of the evidence demonstrating the product’s effectiveness and its safety profile continues to provide compelling evidence supporting an overall favorable benefit-risk profile. However, the integrity of the product testing data used in the development of the product’s manufacturing process is still a matter that we are continuing to evaluate and take very seriously," said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research.

Forum Thread: Novartis Manipulated Zolgensma Data

BioTwitter was is shock, with many respected pundits sharing that they've never seen anything so brazen.

Whether the cover up is worse than the crime is up for debate. Regardless, the optics of the situation are terrible for Novartis. A company the size of Novartis shouldn't put themselves in a situation where they are manipulating data for any drug therapy, let alone one that treats children and costs $2.1 million.

On Wednesday, Novartis responded to the FDA statement and said they “thoroughly, aggressively” investigated the issue and that patient safety was never threatened.

There is a great thread discussing the company's response:

Will physicians be able to trust Novartis again?

Swing over to our Novartis company forum and share your thoughts.