Sen. Chuck Grassley, R-Iowa, the chairman of the U.S. Senate Finance Committee, is the latest lawmaker on Capitol Hill probing what led to Novartis AG submitting falsified data in its U.S. marketing application for its gene therapy Zolgensma and waiting until later to tell the U.S. Food and Drug Administration about it.
Sen. Chuck Grassley
The drug is used to treat children under two years old with spinal muscular atrophy, a group of genetic neuromuscular disorders characterized by the loss of motor neurons and progressive muscle wasting.
Zolgensma is being marketed in the U.S. for $2.1 million, making it the most expensive medicine in American history.
On Aug. 6, the FDA said it had been informed that data in Zolgensma's U.S. marketing application had been manipulated. Novartis had known about the data manipulation since March — two months before the drug won approval — but waited until late June to tell the FDA.
On Aug. 9, a group of senators led by Sen. Dick Durbin, D-Ill., the Senate Democratic whip, called on the FDA to use its full authority to hold Novartis and its subsidiary AveXis Inc. accountable for using the falsified data to obtain approval of Zolgensma.
The lawmakers also wanted to know why the Trump administration withdrew a proposed rule in October 2018 that would have required sponsors of certain clinical trials to promptly report suspected data falsification to the FDA and asked if the agency would reconsider reissuing the regulation in light of the Novartis situation.
In a letter Grassley made public Aug. 12, he told Novartis CEO Vas Narasimhan that his company's conduct — being aware of the data manipulation but intentionally withholding that information from the FDA until after Zolgensma was approved — was "reprehensible" and could have adverse effects on patients.
"Accordingly, the conduct ought to be investigated and, as appropriate, punished to the fullest extent of the law," Grassley wrote.
The Senate Finance Committee chief demanded Novartis turn over certain documents and other information by Aug. 23, including all records related to the decision to withhold information from the FDA about the manipulated Zolgensma data.
Grassley also said he wanted any records related to Novartis' internal inquiry into the Zolgensma data manipulation and when the company opened and closed its probe.
The Iowa Republican told Novartis to also provide the date Novartis actually learned it had submitted manipulated data to the FDA for Zolgensma.
He also asked Novartis whether it had terminated any employees involved in manipulating the Zolgensma data and what steps the company had taken to ensure a similar situation does not occur again.
Grassley's committee oversees the government's Medicare program, which provides healthcare insurance for elderly and disabled Americans, and Medicaid, which covers people with low income.
The U.S. Centers for Medicare and Medicaid Services has been grappling with how to pay for pricey drugs like Zolgensma, the head of the agency acknowledged in May.