Novartis said Wednesday that it delayed informing the FDA that it had uncovered potential data manipulation as part of its filing seeking approval of the gene therapy Zolgensma (onasemnogene abeparvovac-xioi) while it undertook a probe into the matter. The therapy gained approval on May 24 for the treatment of patients under two years of age with spinal muscular atrophy (SMA), although CEO Vas Narasimhan noted that the company received the allegations of data manipulation in mid-March.
"We made the decision to progress our quality investigation prior to informing FDA and other regulatory authorities so that we could provide the best information and technical analysis, which we did promptly on completion on June 28," Narasimhan said. The executive added that the decision to delay notifying regulators was not tied to timing of the drug's approval process.
Earlier this week, the FDA disclosed that it was recently notified by Novartis' AveXis unit that some data submitted as part of the filing seeking approval of Zolgensma may have been manipulated. However, the agency noted that it does not expect the issue to alter the availability of the treatment.
Novartis explained that the data in question were obtained from assays used for initial product testing, noting that they are not currently used for commercial product release. The drugmaker added that "as noted by the FDA, the data in question were a small portion of our overall submission."
Novartis said that it is in the process of "exiting" the scientists responsible for the manipulation and does not believe the issue extends beyond those employees. The drugmaker has reportedly also informed regulators in Europe and Japan, stating that the finding is not expected to affect the development of Zolgensma in those markets.
The news comes after Novartis CEO Vas Narasimhan apologised to employees last year for a $1.2-million contract with Michael Cohen, former attorney of US President Donald Trump, which led to the departure of general counsel Felix Ehrat.
Commenting on the news, Vontobel analyst Stefan Schneider said "the Zolgensma issue just shows how difficult it is to change the culture in Novartis, despite the new CEO having made this a key priority." Meanwhile, Raymond James analyst Steven Seedhouse hinted that the issue could further complicate patient access to the drug based on its $2.1-million cost, particularly "given these optics, which could become more than optics if the data manipulation problem runs deeper."
Novartis acquired rights to Zolgensma, previously known as AVXS-101, under its takeover of AveXis last year for $8.7 billion.
For additional analysis on the drug, read ViewPoints: Novartis' Zolgensma sets the new status quo, and ViewPoints: Payers hopeful that Zolgensma may spark new thinking in gene therapy cost models.
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