UNITED STATES – According to an online news article published by www.the-scientist.com, the FDA is investigating Novartis after learning that the company submitted its application for Zolgensma, a gene therapy drug, with manipulated data.
Novartis Pharmaceuticals gained United States Food and Drug Administration (FDA) approval of Zolgensma, a gene therapy drug used in patients under the age of 2 with spinal muscular atrophy (SMA). The gene therapy drug was approved by the FDA in May 2019 and consists of a one-dose treatment for patients.
The prognosis for children with SMA is grim, and the disorder is the leading cause of death in infants related to a genetic disorder. Zolgensma provides a copy of a necessary gene that can be lifechanging for patients. However, one dose of the gene therapy drug costs $2.1 million, making it the most expensive drug in the world.
In late June 2019, Novartis notified the FDA that the drug application for Zolgensma, which was submitted in early 2019, contained manipulated data. Novartis claims it became aware of the manipulated data from its subsidiary, AveXis, the gene therapy unit responsible for developing and manufacturing the drug.
Reports indicate that AveXis knew about the manipulated data in early March 2019, more than two months before the FDA approved Zolgensma on May 24, 2019. Had the FDA been aware of the manipulated data before May 24, 2019, approval of the gene therapy drug would likely have been delayed. Novartis waited more than one month – from May 24, 2019, to late June 2019 to notify the FDA about the data manipulation. Whether Novartis was aware of the data manipulation sooner or whether only members of AveXis were aware of the manipulated data, is unclear.
According to both Novartis and the FDA, the manipulated data in question does not have any effect on the safety of Zolgensma but rather relates to data derived from animals, and not humans. As such, the FDA believes the drug should remain on the market.
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