While FDA recently said Novartis and AveXis did not reveal data on their $2.1 million gene thaerpy for pediatric spinal muscular atrophy until after it was approved, Novartis is defending its actions.
While FDA recently said Novartis and AveXis did not reveal data on their $2.1 million annual drug for pediatric spinal muscular atrophy until after it was approved, Novartis is defending its actions.
In late May, FDA approved Zolgensma, a gene therapy intended to treat children less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 gene-the most severe form of SMA. SMA is a leading genetic cause of infant mortality.
At the time of its approval, the drug garnered criticism because of its high price tag.
Related: FDA adds warning to Xeljanz
However, following FDA’s approval in late June, the agency was informed by the product’s manufacturer, AveXis Inc., about a “data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA,” FDA said in a statement.
Still, the data manipulation does not impact the safety of the drug. “FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application,” the agency said.
“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,” FDA added.
AveXis became aware of the issue of the data manipulation that created inaccuracies in their Biologic Licensing Application (BLA) before FDA approved Zolgensma, yet did not inform the FDA until after the product was approved, according to FDA.
Novartis, however, defended its actions. AveXis voluntarily self-disclosed to the FDA and subsequently to other health authorities that some data previously submitted to the agency as part of Novartis’s BLA package was inaccurate, Novartis said in a statement.
Related: FDA warns opioid repackers
“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,” Novartis said.
Novartis said an investigation was immediately initiated to “rapidly understand any implications and address the situation,” the pharma maker said. “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 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. “We remain steadfast that this important treatment remain available to pediatric patients with SMA less than 2 years of age.”
Novartis said will “continue to work in close cooperation with FDA to appropriately update our submission and address any quality gaps identified.”
“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. We do not expect this to impact the timing of our ongoing Zolgensma regulatory filings and development programs,” Novartis said.
Read more: How FDA’s insomnia drug warning could impact prescribing