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FDA threatens criminal action against Novartis over faulty data used in application for $2.1 million gene therapy

Key Points
  • The FDA is threatening to take criminal action against Novartis, saying the drugmaker used data it knew was inaccurate in its application for a $2.1 million gene therapy.
  • The agency says the gene therapy should remain on the market, even while it's still assessing the situation.
The Novartis headquarters in Basel, Switzerland.
Arnd Wiegmann | Reuters

The Food and Drug Administration is threatening to take criminal action against Novartis, saying Tuesday the drugmaker used data it knew was inaccurate in its application for a $2.1 million gene therapy that was approved in May.

"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 and reviewed by the FDA," Peter Marks, director of FDA's Center for Biologics Evaluation and Research, said in a statement.

AveXis is the gene therapy subsidiary of Novartis. The FDA said the gene therapy for spinal muscular atrophy should remain on the market, even while it's still assessing the situation.

"The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties," Marks added.

U.S.-traded shares of the Swiss drugmaker tumbled 3.6% in early afternoon trading. Biogen, which also produces a spinal muscular atrophy treatment, was up about 1%.

In May, the FDA approved Novartis' gene therapy, making it the world's most expensive drug. The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11,000 births.

In a statement, Novartis said it continues to be "fully confident in the safety, quality and efficacy of Zolgensma.

"The FDA supports the continued marketing and use of Zolgensma for patients with spinal muscular atrophy (SMA) less than 2 years of age," the statement read.  "We maintain that the totality of the evidence demonstrating the product's effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile. We remain steadfast that this important treatment remain available to pediatric patients with SMA less than 2 years of age."

The company added that, as noted by the FDA, "the data in question were a small portion of our overall submission and are limited to an older process no longer in use." According to Novartis, findings from the investigation did not indicate issues with product safety, efficacy or quality. 

At the time the FDA approved the gene therapy, Acting FDA Commissioner Ned Sharpless lauded the approval, saying that it marked "another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases."

In a tweet Tuesday, Sharpless said the agency relies "on truthful scientific data to make regulatory decisions, and we take the issue of data integrity very seriously."

This story is developing. Check back for updates.