In May, the U.S. Food and Drug Administration (FDA) approved Novartis’ gene therapy Zolgensma as a one-time treatment for spinal muscular atrophy. On Monday though, the regulatory agency revealed that data manipulation was involved in the preclinical process but suggests that the therapy remain on the market.
In an announcement Monday, the FDA said on June 28, more than a month after the agency approved the gene therapy, AveXis Inc., a Novartis subsidiary, informed the FDA “about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application.” The FDA said it is carefully assessing the information and situation, but “remains confident” that Zolgensma should remain on the market.
The FDA said its concerns are “limited to only a small portion of the product testing data that was contained in the marketing application.” That testing data was used by AveXis to support the development of its production process, the FDA said, but added that the data does not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program.
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