Novartis Concealed Manipulated Data From FDA While Seeking Approval For $2.1M Gene Therapy Drug
Officials say the issue doesn't put patients at risk, but the drugmaker could face penalties for withholding the information. The news has also unsettled an industry where many are racing to be the first to come out with these expensive gene therapies.
The Associated Press:
FDA Says Novartis Withheld Data Problem Before Drug Approval
U.S. regulators want to know why Novartis didn't disclose a problem with testing data until after the Swiss drugmaker's $2.1 million gene therapy was approved. The Food and Drug Administration said Tuesday the manipulated data involved testing in animals, not patients, and it's confident that the drug, called Zolgensma, should remain on the market. The agency said it's investigating and will consider criminal or civil penalties if appropriate. (Johnson, 8/6)
The New York Times:
Novartis Hid Manipulated Data While Seeking Approval For $2.1 Million Treatment
Officials said the inaccurate data, which involved testing in mice of two different strengths of the treatment, did not affect the safety or efficacy of the therapy, Zolgensma, used to treat a rare, often fatal genetic disease called spinal muscular atrophy. Approved in May, the treatment’s price — set at $2.1 million — stoked concerns about the astronomical costs of potential cures for rare diseases and upset parents who initially could not get insurance coverage for the breakthrough treatment. The F.D.A. said patients were not at risk, and that the treatment could still be sold. (Thomas, 8/6)
The Washington Post:
FDA Says Data Manipulated In Testing Of Novartis’ $2 Million Gene Therapy
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said the inaccurate information involved mouse data and was a “small amount of the totality” that was submitted. The faulty data didn’t raise concerns about the safety and effectiveness of the treatment, he said. But he added the agency takes data integrity issues seriously and is continuing to investigate the situation. He said the FDA “will use its full authorities to take action, if appropriate, which may include civil or criminal penalties” against the manufacturer, AveXis Inc., a subsidiary of Novartis. (McGinley, 8/6)
The Wall Street Journal:
FDA Says Data Manipulated For Novartis Gene Therapy Drug
“At no time during the investigation did the findings indicate issues with product safety, efficacy or quality,” the company said, adding that it “is committed to taking appropriate action to prevent future incidents across its portfolio of development programs.” The company in the past has defended the lofty price by comparing it to another treatment that Novartis said would cost twice as much over a 10-year period. (Burton, 8/6)
USA Today:
Novartis Used Faulty Data For World's Most Expensive Drug Zolgensma
But officials warned that AveXis, the gene therapy subsidiary of Swiss giant Novartis, could face penalties. "We rely on truthful scientific data to make regulatory decisions, and we take the issue of data integrity very seriously," FDA Commissioner Ned Sharpless said on Twitter. "In this case, the agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties." (Alltucker, 8/6)
Stat:
Novartis Faces A New Crisis. For CEO Vas Narasimhan, This Time Is Different
Narasimhan, 43, took the reins at Novartis with the stated goal of changing the culture at a drug company that traces its roots back to 1859. In more recent years, Novartis has faced a string of allegations of bribery, charges of data falsification, and accusations of illegal marketing. Narasimhan promised to transform the company and make such scandals a thing of the past. (Garde, 8/7)
Reuters:
U.S. FDA Says Some Data Testing Novartis' $2 Million Gene Therapy Was Manipulated
Novartis acquired the therapy’s maker in 2018, and the drugmaker was aware of the manipulation as early as March - more than two months before the treatment’s approval, the FDA said. (Erman, 8/6)
