On Aug. 6, 2019, the U.S. Food and Drug Administration (FDA) issued a statement saying that Swiss pharmaceutical company Novartis AG submitted a biologics license application (BLA) for the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi) with manipulated data and failed to inform regulators until June 28, 2019—more than a month after the FDA approved the drug.1 2
According to Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research (CBER), Novartis subsidiary company AveXis, Inc. Bannockburn, Illinois “became aware of the issue of the data manipulation that created inaccuracies in their BLA before the FDA approved the product, yet did not inform the FDA until after the product was approved.”1
Dr. Marks added, “The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties.”1
A recent article by FiercePharma noted that the revelation regarding Zolgensma data manipulation is “only the latest regulatory misstep for Novartis, which has been working to bolster its compliance following a number of scandals around the world in recent years. From 2013 to 2015, scandals erupted in Japan as authorities discovered Novartis manipulated data and failed to report side effects.”5
“[Novartis] has also been hit with bribery allegations in various countries,” reported FiercePharma. One of these scandals involves an eight-year-old whistleblower lawsuit alleging that Novartis bribed doctors in the U.S. to boost subscriptions of its prescription drugs, including the hypertension drug Lotrel (amlodipine besylate and benazepril hydrochloride) and the diabetes drug Starlix (nateglinide).5 6
Novartis has reportedly set aside $700 million for a possible settlement of this case.6
Up until 2014-2015, Novartis was a major manufacturer of vaccines. In 2014, the company was still considered among the top five vaccine makers, along with Merck, Sanofi Pasteur, GlaxoSmithKline (GSK) and Pfizer. By the end of 2015, however, Novartis had sold off its vaccine operations to CSL Ltd. of Melbourne, Australia and the U.K.’s GSK and was out of the market altogether.7 8 9 10
1 Marks P. Statement on data accuracy issues with recently approved gene therapy. U.S. Food and Drug Administration Aug. 6, 2019.
2 ZOLGENSMA® (onasemnogene abeparvovec-xioi). Zolgensma.com.
3 SMI Identified. Invitae Corporation.
4 Genetics Home Reference. Spinal muscular atrophy. U.S. National Library of Medicine.
5 Sagonowsky E. FDA ready to crack down as Novartis reveals manipulated Zolgensma data. FiercePharma Aug. 6, 2019.
6 Blankenship K. Novartis, aiming to clean up its rep, sets aside $700M for kickback settlement. FiercePharma July 18, 2019.
7 Cáceres M. Merck Leads World’s Top Vaccine Makers, Novartis Out. The Vaccine Reaction Aug. 28, 2015.
8 GSK Pharma completes acquisition of Novartis Healthcare’s vaccines business. Pharmaceutical Technology Sept. 30, 2015
9 Helfand C. Novartis bids farewell to vaccines with $7.1B sale to GSK. FiercePharma Apr. 22, 2014.
10 Sagonowsky E. CSL completes purchase of Novartis’ flu vaccine business. FiercePharma Aug. 3, 2015.
11 Roos R. Novartis flu vaccine on hold in Italy after suspicious deaths. Center for Infectious Disease Research and Policy Dec. 1, 2014.