The US Food and Drug Administration (FDA) announced August 6 that it was recently informed by AveXis, the developer of Zolgensma, about a manipulation issue regarding the drug that “impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA).”
Zolgensma (onasemnogene abeparvovec-xioi) is a gene therapy indicated for the treatment of spinal muscular atrophy (SMA) in infants, for which Novartis recently received approval after acquiring AveXis in 2018.
Novartis has priced the therapy at $2.1m (€1.88m) per patient, making it the most expensive treatment on the market.
However, the regulatory agency stated that the product will remain on the market, as the concerns are limited to “only a small portion of the product testing data” contained in the marketing application, adding that the positive assessment on the drug remains in place.
AveXis, according to the FDA’s announcement, became aware of the data manipulation before the approval, yet did not inform the FDA until after the product was approved.
The FDA stated that it will “take action, if appropriate,” with civil or criminal penalties against the company.
In an answering announcement, Novartis said that it is ‘fully confident’ in the safety, quality and efficacy of the drug, claiming that the evidence supports an “overall favorable benefit-risk profile.”
According to Novartis, on June 28th AveXis voluntarily self-disclosed that some data submitted as part of our BLA was inaccurate, as it “had become aware of allegations of data manipulation in a specific animal testing procedure.”
Novartis states that the assays in question were used for initial product testing and are not currently used for commercial product release.
The company also states that it initiated an investigation to understand any implications and shared the findings with the FDA, which however did not indicate issues with product safety, efficacy or quality.
Ultimately, Novartis notes that it will work to update the BLA submission and “address any quality gaps identified.”