The U.S. Food and Drug Administration has opened a probe into Novartis AG's Zolgensma after the company's drug manufacturing unit reported a data manipulation issue related to animal testing that was done to support the gene therapy's approval.
Zolgensma was approved in May to treat spinal muscular atrophy, a rare genetic childhood disease that impacts the nervous system that controls voluntary movement. The drug was priced at $2.1 million, making it the most expensive therapy in the world.
The U.S. regulator was informed June 28 by Novartis unit AveXis Inc. about the data manipulation issue that "impacts the accuracy" of data collected from testing the product in animals. Specifically, the data was used to support the development of the production process for Zolgensma.
Moreover, the FDA said that AveXis was aware of the inaccurate data before the drug was approved, but the agency was not informed.
"The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties," according to the FDA's statement.
However, the agency said the data issues do not impact the drug's benefits shown during clinical trials.
"These data do not change the agency's positive assessment of the information from the human clinical trials that were conducted as part of the development program," the agency said in an Aug. 6 statement. The FDA "remains confident that Zolgensma should remain on the market."
But the FDA is still taking the data issue seriously. The agency conducted an inspection of an AveXis facility in California from July 24 to Aug. 2, finding that data derived from a number of studies may have been mismanaged or manipulated. Laboratory records were also missing information on compliance measures and some information on early mouse studies was delivered verbally, rather than recorded as required.
Leerink said in a note issued shortly after the FDA's statement that the disclosure may increase regulators' scrutiny on the manufacturing process for Zolgensma — from the FDA to the EU's European Medicines Agency. The issues flagged by the regulator do, however, appear "addressable," according to Leerink.
Shares of Novartis dropped nearly 3% to $88.02 as of 2:33 p.m. ET after the FDA issued its statement.
Novartis responded to news of the investigation later the same day, stating that it would stand behind the gene therapy.
"[W]e are fully confident in the safety, quality and efficacy of Zolgensma," Novartis said in its Aug. 6 news release. "We maintain that the totality of the evidence demonstrating the product's effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile."
Novartis said that allegations of data manipulation had been self-reported by Avexis during animal testing and were part of an older process no longer in use.
"We are committed to ensuring the highest levels of transparency and integrity with health agencies, as well as with the patients and providers we serve," Novartis said, adding that it does not expect the regulatory and development timing was not expected to change.