FDA says some data used to approve world's most expensive drug was faulty

FDA says some data used to approve world's most expensive drug was faulty
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The world’s most expensive drug was approved using data that was partially faulty, the Food and Drug Administration (FDA) said Tuesday. 

The drug, Zolgensma, was approved in May at the sky-high price of $2.1 million. It treats the deadly childhood disease spinal muscular atrophy. 

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Now, the FDA is saying it has learned that some of the product testing data used to approve the drug was inaccurate. 

Furthermore, the FDA said the company that makes the drug, AveXis, a unit of Novartis, learned the data was incorrect before the drug was approved, but waited until after approval to tell the FDA. 

“The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties,” Peter Marks, an FDA official, said in a statement. 

Despite the problems with some of the data, the FDA said it “remains confident that Zolgensma should remain on the market.”

The FDA said “only a small portion” of the product testing data was faulty due to a “data manipulation issue.”

The revelation is sure to increase scrutiny of Novartis, which was already facing criticism from drug pricing advocates for the price of the drug. 

"We are fully confident in the safety, quality and efficacy of Zolgensma," Novartis said in a statement.

"We have and will continue to work in close cooperation with the FDA to appropriately update our submission and address any quality gaps identified," the company added. "We are committed to ensuring the highest levels of transparency and integrity with health agencies, as well as with the patients and providers we serve."

The company said that once it learned of the allegations of data manipulation "an investigation was immediately initiated to rapidly understand any implications and address the situation." 

"Once we had interim conclusions from our investigations, we shared our findings with the FDA," the company said.  

“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,” Marks said. “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."