FDA Assessing Data Accuracy in Zolgensma Approval

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The agency is keeping Zolgensma on the market as it evaluates the accuracy of data submitted with the product’s biologics license application.

FDA announced on August 6, 2019 that it was assessing the accuracy of certain animal testing data submitted in the biologics license application (BLA) of Zolgensma. The gene therapy was approved by FDA in May 2019 and is used to treat children less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene.

On June 28, 2019, AveXis Inc., a Novartis Company, the product’s manufacturer, informed FDA about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the BLA reviewed by FDA. The company was aware of the data manipulation before approval of the drug, according to FDA, but did not inform the agency until after the product was approved. “The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties,” FDA stated in a press release.

According to the agency, the data affected were a small portion of the information submitted in the BLA. The data were used to support the development of the company’s production process and do not change FDA’s positive assessment of the human clinical trials conducted in the development program. “We are carefully assessing the issue of the manipulation of the product testing data used in the production process and are conducting a thorough assessment of the information from a recently completed inspection.  In part, this will allow us to determine the implications for the FDA’s scientific review of the information in the BLA and will allow us to amend our publicly posted BLA reviews, as appropriate,” the agency stated.


“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. 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,” the agency stated in the press release.

Source: FDA