According to the Food and Drug Administration (FDA), AveXis Inc, the manufacturer of the gene therapy Zolgensma, was aware of an issue related to “data manipulation” prior to approval, but did not inform the Agency until after the product was approved.
In a press release, Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research stated that the data manipulation issue impacts the accuracy of certain product testing data presented in the Biologics License Application (BLA) for Zolgensma, a recombinant adeno-associated virus vector-based gene therapy indicated for the treatment of children with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The FDA noted, however, that the issue does not impact the positive assessment it has with regard to the product’s effectiveness based on human clinical trials.
“The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market,” said Marks. “The totality of the evidence demonstrating the product’s effectiveness and its safety profile continues to provide compelling evidence supporting an overall favorable benefit-risk profile. However, the integrity of the product testing data used in the development of the product’s manufacturing process is still a matter that we are continuing to evaluate and take very seriously.”
In response to the FDA, AveXis’ parent company Novartis issued a statement addressing the data integrity issues:
“On June 28th, AveXis voluntarily self-disclosed to the FDA and subsequently to other health authorities that some data previously submitted to the agency as part of our BLA package was inaccurate. Avexis had become aware of allegations of data manipulation in a specific animal testing procedure used in the development of the product. The assays in question were used for initial product testing and are not currently used for commercial product release. An investigation was immediately initiated to rapidly understand any implications and address the situation. Once we had interim conclusions from our investigations, we shared our findings with the FDA. As noted by the FDA, the data in question were a small portion of our overall submission and are limited to an older process no longer in use.”
The Company stated that it is fully cooperating with the Agency to address any gaps in the BLA.
“We are carefully assessing the issue of the manipulation of the product testing data used in the production process and are conducting a thorough assessment of the information from a recently completed inspection,” said Marks, adding that the FDA “will use its full authorities to take action” which he states may include “civil or criminal penalties.”
Healthcare professionals with questions related to Zolgensma can contact the FDA at (800) 835-4709.
For more information visit fda.gov.