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Novartis hid faulty data from FDA over $2.1 million gene therapy Zolgensma

Published on 07/08/19 at 09:55am

Novartis hid faulty data from the FDA until their drug was approved, according to a statement from Dr Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER).

Novartis subsidiary AveXis failed to inform the FDA of inaccuracies in the data for $2.1 million gene therapy Zolgensma, until after the regulator had approved the drug, despite being aware of the fault.

While the FDA has said that they are confident that “Zolgensma should remain on the market,” the agency warned it “will use its full authorities to take action, if appropriate, which may include civil or criminal penalties.”

The scandal comes after the US regulator approved spinal muscular atrophy (SMA) drug Zolgensma on 24 May 2019. On 28 June, AveXis told the FDA that there had been a data manipulation issue that impacts the accuracy of some of the data taken from product testing in animals. However the firm had been aware of the innacuracies before the drug had been approved.

The FDA’s concerns are thus limited to just a small portion of the product testing data that was contained in the marketing application. Yet the scandal has called Novartis' integrity into question.

Dr Marks commented in a statement: “Ensuring truthful, complete and accurate data in product applications is a critical component of industry’s responsibility as they work to demonstrate the safety, purity, and potency of biological products. The submission of such truthful, complete and accurate data is also critical for the FDA to be able to protect the public health, and the law requires it.”

Shares in Novartis fell by 3.6% on announcement of the news, as the Swiss drugmaker sought to reassure the public that they “are fully confident in the safety, quality and efficacy of Zolgensma.”

Novartis added: “We have and will continue to work in close cooperation with the FDA to appropriately update our submission and address any quality gaps identified. We are committed to ensuring the highest levels of transparency and integrity with health agencies, as well as with the patients and providers we serve.”

Louis Goss

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