Five Democratic senators – two of them presidential candidates – are calling on the head of the Food and Drug Administration to take action in the wake of revelations last week that one of the world’s largest drugmakers had submitted manipulated data to the agency in its approval application for a gene therapy.
In the letter, sent Friday to acting FDA Commissioner Ned Sharpless, the senators wrote that the scandal that emerged last week when the agency revealed allegations that certain animal testing data for Swiss drugmaker Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec-xioi) were manipulated, but that the company did not inform the agency until after it was approved, highlights what they called the drug industry’s “privilege and greed.” The letter was signed by Sens. Dick Durbin, Illinois, Tammy Baldwin, Wisconsin, Bernie Sanders, Vermont, Elizabeth Warren, Massachusetts, and Richard Blumenthal, Connecticut.
“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,” they wrote. “Such greed cannot be condoned by the FDA.”
Zolgensma – originally developed by AveXis, which Novartis acquired for $8.7 billion last year – received FDA approval in May for spinal muscular atrophy, and was given a $2.1 million list price.
A Novartis spokesperson pointed to a statement the company released last week, standing by the therapy.
However, the larger issue is not Zolgensma’s benefit-risk profile. Indeed, FDA officials have recommended it remain on the market. Rather, it’s that the company allegedly knew of the data manipulation issue in March, but did not tell the agency until late June, more than a month after Zolgensma’s approval. The company said that upon learning of the problem in the middle of March, it launched a two-part investigation.
During a conference call with analysts last week, when asked when the news landed on his desk, CEO Vas Narasimhan did not answer directly, though the spokesperson wrote in an email that he found out in May.
But in the letter Friday, the senators noted that federally funded research by the National Institutes of Health had contributed to Zolgensma’s development, and that the company received taxpayer-funded benefits designed to expedite the approval process like Fast Track, Breakthrough and Priority Review designations. In response, they urged the agency to use its authority to hold AveXis accountable, including through criminal, civil and regulatory actions. The FDA had stated last week the civil and possible criminal penalties were possible.
They also sought a formal, written explanation for why the agency had withdrawn a proposed regulation in October 2018 that would have required certain clinical trial sponsors to promptly report suspected data falsification to the agency. “In light of AveXis’ alarming actions, we also seek to know whether FDA plans to re-issue such regulation,” they wrote.
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