Drug vials AveXis Inc., thesubsidiary that manufactures Zolgensma, brought a “datamanipulation issue” related to testing performed on animals to theFDA’s attention five weeks after the drug’s approval. (Photo:Shutterstock)

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Novartis made headlines earlier this year withthe debut of Zolgensma, the most-expensive drugto receive FDA approval to date.

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Now, the drugmaker and its $2.1 million treatment are back inthe headlines–this time facing potential criminalcharges after the FDA said that Novartis withheld information aboutan error in testing data until after the drug was approved.

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Related: Staggering prices slow insurers’ coverage of genetherapy

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The drug was approved on May 24, and it was about another monthbefore the company notified the FDA of the problem with thedata.

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Zolgensma is a gene therapy used to treat the childhood diseasespinal muscular atrophy. According to astatement, the FDA “remains confident that Zolgensmashould remain on the market,” even though Peter Marks, an FDAofficial, adds that “[t]he agency will use its full authorities totake action, if appropriate, which may include civil or criminalpenalties.”

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As reported by the Associated Press, AveXis Inc., the Novartissubsidiary that manufactures Zolgensma, brought a “datamanipulation issue” related to testing performed onanimals to the FDA’s attention five weeks after the drug’sapproval. Although Novartis says the animal test is not tobe used in making the therapy for patients, the company did notaddress the issue of why the inaccuracy was not immediatelyreported to the FDA.

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The FDA staff also found several deficiencies, including afailure to completely follow quality control procedures, in arecent inspection of the company’s Irvine, California,manufacturing plant.

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“The totality of the evidence demonstrating the product’seffectiveness and its safety profile continues to providecompelling evidence supporting an overall favorable benefit-riskprofile,” Marks says. ”However, the integrity of theproduct testing data used in the development of the product’smanufacturing process is still a matter that we are continuing toevaluate and take very seriously.”

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Emily Payne

Emily Payne is director, content analytics for ALM's Business & Finance Markets and former managing editor for BenefitsPRO. A Wisconsin native, she has spent the past decade writing and editing for various athletic and fitness publications. She holds an English degree and Business certificate from the University of Wisconsin.