- Sen. Chuck Grassley, the Republican chairman of the powerful Senate Finance Committee, is demanding a full accounting from Novartis of its investigation into faulty data it submitted to the Food and Drug Administration in seeking approval for the gene therapy Zolgensma.
- In particular, Grassley seeks records from Novartis related to its internal inquiry into the data manipulation, as well as any tied to its decision to delay informing the FDA until after the agency had approved Zolgensma.
- “Such conduct is reprehensible and could have an adverse effect on patients,” Sen. Grassley wrote in a letter dated August 9. “Accordingly, the conduct ought to be investigated and, as appropriate, punished to the fullest extent of the law.”
Grassley, R-Iowa, isn’t the first lawmaker to take interest in the revelation Novartis used manipulated data in winning clearance for Zolgensma (onasemnogene abeparvovec). Five Democratic senators, including presidential candidates Bernie Sanders, I-Vt., and Elizabeth Warren, D-Mass., wrote the FDA last week complaining of Novartis’ “unscrupulous action.”
Grassley’s role as chairman of the Senate Finance Committee, however, gives his inquiries more weight.
In addition to seeking records, Grassley wants information on when Novartis learned of the data inaccuracies, as well as when it opened and closed its investigation into the manipulation. Of note, the lawmaker is also interested in the steps Novartis has taken since to ensure faulty data isn’t included in future submissions to the FDA.
The letter sets a deadline of August 23 for Novartis to reply.
Some of the timeline surrounding the manipulation and Novartis’ response is already known. The manipulation occurred in testing conducted in mice to compare versions of Zolgensma used in Phase 1 and Phase 3 trials.
An FDA inspection report found an initial allegation of potential data mismanagement was made on March 14 to the chief quality officer of AveXis, the biotech developer of Zolgensma that Novartis bought last year. Two months later, on May 15, a non-conformance report was opened to investigate the claim.
Novartis CEO Vas Narasimhan, explaining the company’s response on a call with investors, indicated an initial independent investigation was conducted between mid-March and early May. When the data discrepancies were substantiated in May, Novartis then opened a full technical quality investigation that yielded findings on June 28, the same day the company informed the FDA.
In the interim, on May 24, the regulator had cleared Zolgensma as a treatment for spinal muscular atrophy, a rare neurodegenerative disease that typically results in death in its most severe form.
Novartis priced Zolgensma at $2.1 million per dose, making it the most expensive drug ever brought to market and a lightning rod for criticism of sky-high drug prices.
In an internal memo, a senior FDA official noted the agency would have delayed its decision if it had known of the data manipulation sooner, although he indicated his belief Zolgensma would still have eventually been approved. Still, the FDA has affirmed that the treatment should remain on the market.
Disclosure of the data issues raised questions of whether Novartis deliberately delayed relaying its findings to the FDA in order to ensure an on-time approval for Zoglensma —? a conclusion CEO Narasimhan strongly denied.
“We made the decision to progress our quality investigation prior to informing FDA and other regulatory authorities so that we could provide the best information and technical analysis, which we did promptly on completion on June 28,” he said on the call to investors.
That hasn’t taken any heat off the drugmaker though.
“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,” wrote the five Democratic senators in their August 9 letter to acting FDA Commissioner Ned Sharpless.
Novartis has received and is currently reviewing Grassley’s letter, a spokesperson for the company confirmed.