The US Food and Drug Administration says some data from early testing of Novartis' Zolgensma (onasemnogene abeparvovec-xioi) was manipulated, but the agency says the gene therapy should remain on the market, Reuters reports.
FDA approved Zolgensma in May to treat children under the age of two years shown by genetic testing to have a certain form of spinal muscular atrophy. The one-time treatment, which delivers a working form of the SMN gene, has a list price of $2.1 million.
In a statement, the FDA says AveXis, which was acquired by Novartis in 2018, informed it about a data manipulation issue that could affect the accuracy of the data from the drug's testing in animals that was used to support its biologics license application. It does not, the agency says, affect its evaluation based on human clinical trials. Still, FDA adds that it "is carefully assessing this situation."
Novartis says in its own statement that it is "fully confident in the safety, quality, and efficacy of Zolgensma."
The Wall Street Journal notes, though, the company reported the issue to the FDA after the drug had been approved, even though it was aware of the problem as early as March. The regulators say that had they known earlier, the approval of the drug likely would still have occurred, though after a delay, it adds.
Peter Marks, the director of FDA's Center for Biologics Evaluation and Research, says in the statement that the "agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties."