The US Food and Drug Administration (FDA) has issued a statement regarding data accuracy issues related with Novartis鈥� 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鈥檚 statement, a month following Zolgensma鈥檚 approval, the gene therapy鈥檚 manufacturer AveXis informe the FDA there was a 鈥渄ata manipulation issue鈥� impacting the 鈥渁ccuracy of certain data from product testing performed in animals鈥�, which composed part of the BLA.
The FDA continued in the statement, saying: 鈥淭he totality of the evidence demonstrating the product鈥檚 effectiveness and its safety profile continues to provide compelling evidence supporting an overall favourable benefit-risk profile.
鈥淗owever, the integrity of the product testing data used in the development of the product鈥檚 manufacturing process is still a matter that we are continuing to evaluate and take very seriously.鈥�
In a statement Novartis echoed the FDA鈥檚 sentiment, saying 鈥渨e are fully confident in the safety, quality and efficacy of Zolgensma鈥e maintain that the totality of the evidence demonstrating the product鈥檚 effectiveness and its safety profile continue to provide compelling evidence supporting an overall favourable benefit-risk profile.鈥�
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By GlobalDataIt 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 鈥渢ruthful, complete and accurate data in product applications鈥� 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 鈥渁n 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.鈥�
鈥淎t no time during the investigation did the findings indicate issues with product safety, efficacy or quality.鈥�
