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Senators attack Novartis over 'unconscionable' handling of Zolgensma data

Ask FDA to hold the company ‘accountable’


US senators, including potential presidential candidates Bernie Sanders and Elizabeth Warren, have attacked Novartis over its handling of gene therapy data.

Novartis’ filing for approval of spinal muscular atrophy gene therapy Zolgensma was thrust into the spotlight last week when the US Food and Drug Administration (FDA) issued a statement about a “data manipulation issue”. The allegations centre on the behaviour of AveXis, which Novartis bought for $8.7bn before the gene therapy company filed for approval of Zolgensma.

In the FDA’s telling of events, AveXis knew about the manipulation of animal testing data included in its submission before Zolgensma was approved but only told the agency after receiving marketing authorisation in the US.

Novartis has defended itself against the allegations, stating it is “fully confident in the safety, quality and efficacy of Zolgensma”, but now faces an escalating backlash led by a handful of US senators.

The senators set out their concerns in a public letter to FDA Acting Commissioner Ned Sharpless.

“It is unconscionable that a drug company would provide manipulated data to federal regulators in order to rush its product to market, reap federal perks and charge the highest amount in American history for its medication. Such greed cannot be condoned by the FDA. This scandal smacks of the pharmaceutical industry’s privilege and greed, and Americans are sick of it,” the senators wrote.

Political interest in the Zolgensma filing is fuelled by the price Novartis set for the gene therapy. At $2.1m, the rare disease gene therapy is the most expensive drug in American history.

As Sanders, Warren and their co-authors see it, AveXis failed to disclose the data issue because of its rush to start selling the gene therapy at that price.

The senators think AveXis’ behaviour is particularly egregious in light of the US federal support it received while bringing Zolgensma to market. The letter highlights the fast track, breakthrough and priority review perks the FDA granted Zolgensma.

AveXis also received a rare paediatric priority review voucher from the FDA upon approval of its gene therapy, which it can either sell or use to cut the time it takes to bring another drug to market. Earlier this year, Biohaven Pharmaceutical paid $105m for a rare paediatric priority review voucher.

The involvement of high-profile senators in the case could have implications beyond Novartis. One thing the senators want to know is why the FDA withdrew a proposed regulation covering the prompt reporting of suspected data falsification.

Article by
Nick Taylor

12th August 2019

From: Regulatory



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