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Novartis defends Zolgensma as FDA investigates ‘data manipulation’

Gene therapy will remain on the market


Novartis withheld data prior to the approval of Zolgensma, its spinal muscular atrophy (SMA) gene therapy, the US Food and Drug Administration (FDA) has alleged. 

According to a statement issued by the FDA on 6 August, the agency was made aware of a “data manipulation issue” by the manufacturer of Zolgensma, AveXis Inc. Novartis acquired the gene therapy specialist AveXis in April last year and Zolgensma had been subsequently developed by them.

The FDA also said that AveXis was aware of this issue prior to the FDA approval of the drug, but did not inform the agency until 28 June, after the drug won this approval. The data withheld from the FDA allegedly impacts the accuracy of certain data from product testing performed on animals, and was subsequently submitted in the license application which was reviewed by the FDA.

Despite this issue, the FDA has said that Zolgensma should remain on the market while the situation is investigated. They maintain that the data issue affects only a small amount of the product testing data within the marketing application for the drug. There is no indication that human study results were manipulated, with the manipulated data only affecting some findings from the earliest stages of human studies.

Novartis has hit back against the announcement from the FDA, maintaining that it is “fully confident in the safety, quality and efficacy of Zolgensma”. The Swiss pharma company also maintained that it immediately began an investigation into the allegation of data manipulation and shared findings with regulators, including the FDA.

However, the FDA has said that the company could face civil or criminal penalties as a result of withholding the data.

Zolgensma was approved in the US earlier this year, with Novartis setting its price at $2.12m per patient. This made it the first US drug costing over a million dollars and the world’s most expensive medicine.

It was also the first gene therapy approved for paediatric patients under two years of age with SMA, which is a rare genetic disorder that has long been a death sentence for children suffering with the condition.

There had already been concerns over how commercially sustainable the field of cell and gene therapy is, with Zolgensma’s price point having raised eyebrows when it was first approved.

This announcement could again cause concerns – although shares were only slightly changed, falling to 3% in Zurich trading on Wednesday morning.

The FDA has said it will continue to provide updates as the situation continues, while Novartis has said that it will cooperate with the agency to ensure any quality gaps are addressed in full. The pharma company does not anticipate that this situation will impact the timing of ongoing regulatory filings and development programmes involving Zolgensma.

Article by
Lucy Parsons

6th August 2019

From: Regulatory



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