Novartis Knew of Test Data Manipulation Before Drug Approval, FDA Says

Drug giant Novartis knew of allegations of data manipulation in the testing process for a drug — even before the U.S. Food and Drug Administration (FDA) finalized the approval, the agency announced Tuesday.

Yet both the Swiss pharmaceutical giant and the FDA support keeping Zolgensma on the market — even as the federal regulator said it was investigating civil, potentially criminal, penalties.

“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,” said Peter Marks, M.D., Ph.D., the director of the Center for Biologics Evaluation and Research, in an FDA statement Tuesday afternoon.

“We are also aware that AveXis (the Novartis subsidiary) became aware of the issue of the data manipulation that created inaccuracies in their (biologics license application) before the FDA approved the product, yet did not inform the FDA until after the product was approved,” added Marks. “The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties.”

Zolgensma, approved by the FDA in May, is generally described as the “most expensive” treatment currently offered in medicine. A single dose of the gene therapy costs over $2 million. It treats a rare inherited condition called spinal muscular atrophy (SMA), which is caused by mutations in the survival motor neuron 1 gene, which impacts muscle control and is a leading cause of infant mortality. Varying degrees of SMA affect approximately 1 in 11,000 babies each year, according to the advocacy group Cure SMA.

Novartis said in its own statement Tuesday that the data manipulation was made in “a specific animal testing procedure used in the development of the product” — and that it shared its “interim findings” of an initial investigation with the FDA when they were available.

“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,” according to the pharma giant’s statement. “We remain steadfast that this important treatment remain available to pediatric patients with SMA less than 2 year of age.”

The next actions by the FDA remain unclear, the company said.

“We rely on truthful scientific data to make regulatory decisions, and we take the issue of data integrity very seriously,” tweeted Ned Sharpless, M.D., the acting FDA Commissioner, on Tuesday. “In this case, the agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties."

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