Novartis Pledges to Do Things Differently for FDA, After Zolgensma Controversy

Seth Augenstein
SEPTEMBER 10, 2019
Pills, hands, fda,

Novartis knew about allegations of data manipulation in one of its pending drug approvals before the U.S. Food and Drug Administration (FDA) took final action in May. It was only after the drug received the federal okay that the pharmaceutical giant told the federal regulator of the scientific problems.

But in an investor meeting Monday, the head of the company pledged transparency in such situations in the future – even as he contended Novartis did nothing wrong in the 30-plus working days the company investigated internally the testing of Zolgensma.

“Going forward, though, we’ve learned from this,” said Vas Narasimhan, M.D., the Novartis CEO, during the webcast of the London meeting. “What we’ve decided to do is voluntarily and proactively taken a pledge with the FDA to ensure that we will inform them within five business days of any credible allegation of a data integrity issue of a filing in the Novartis group.”

The data manipulation involved trials using mouse cage cards, according to Narasimhan.

The company believes the data issues arose prior to Novartis acquiring AveXis, the subsidiary that developed Zolgensma, he added.

Safety, efficacy, and quality were not affected in the data changes, the CEO said at the Monday meeting.

Action had been taken against “those individuals we believe are responsible,” Narasimhan added.  

The full investigation took more than 30 working days – and the results have been laid out in a FDA Form 483 inspection document, which has not yet been made public, according to the CEO.

Despite the data manipulation, both Novartis and federal regulators are in support of keeping the treatment on the market.

Zolgensma, approved by the FDA in May, is generally described as the “most expensive” treatment currently offered. 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.

However, the FDA said last month that the pharma giant’s actions leave potential legal recourse open.

“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,” said Peter Marks, M.D., Ph.D., the director of the Center for Biologics Evaluations and Research. “The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties.”

Immediately after the Zolgensma data manipulation was revealed, Novartis stock dipped. But within a week, the Swiss pharma giant’s price had recovered. Later reports indicated that a Novartis insider sold nearly $1 million of shares just weeks before the Zolgensma allegations were made public

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