The US Food and Drug Administration (FDA) has issued a statement regarding data accuracy issues related with Novartis’ biologic license application (BLA) for gene therapy Zolgensma (onasemnogene abeparvovec-xioi).

Zolygensma was approved in the US in May this year for children under two years old with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene; this is the most severe form of SMA.

According to the FDA’s statement, a month following Zolgensma’s approval, the gene therapy’s manufacturer AveXis informe the FDA there was a “data manipulation issue” impacting the “accuracy of certain data from product testing performed in animals”, which composed part of the BLA.

The FDA continued in the statement, saying: “The totality of the evidence demonstrating the product’s effectiveness and its safety profile continues to provide compelling evidence supporting an overall favourable 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.”

In a statement Novartis echoed the FDA’s sentiment, saying “we are fully confident in the safety, quality and efficacy of Zolgensma…We maintain that the totality of the evidence demonstrating the product’s effectiveness and its safety profile continue to provide compelling evidence supporting an overall favourable benefit-risk profile.”

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It explained that the assays relevant to the data issues reported by AveXis to the FDA and other healthcare authorities were used in early testing, but have not been included in commercial products.

However, the regulator noted that since “truthful, complete and accurate data in product applications” is the responsibility of the pharmaceutical companies and its partners, it was very concerned that AveXis was aware of the issue of data manipulation before the submission of a BLA for Zolgensma and yet did not inform the FDA, and therefore, it may consider taking civil or criminal action.

Novartis claimed in its statement that “an investigation was immediately initiated o rapidly understand any implications and address the situation. Once we had interim conclusions from our investigations, we shared our findings with the FDA.”

“At no time during the investigation did the findings indicate issues with product safety, efficacy or quality.”