According to documents and interviews reported in The Wall Street Journal, the FDA is focusing on why Novartis took two months to open up a formal inquiry into a test data manipulation issue involving its spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovac-xioi). "There is no documentation," FDA investigators Scott Ballard and Mihaly Ligmond wrote in a recent report, as to why the drugmaker's internal probe "was not opened until May 15 when the initial allegation [of data manipulation] is documented as having been reported on March 14."
CEO Vas Narasimhan has acknowledged that Novartis was aware of the problem ahead of FDA approval of Zolgensma on May 24. Still, both the FDA and Novartis have said that even if it had been known, the data manipulation issue, which involved a small portion of the product testing data, would not have affected the agency's final decision on Zolgensma. The company gained the gene therapy, which costs $2.1 million for a single intravenous infusion, as part of its purchase of AveXis last year for $8.7 billion.
According to a statement to The Wall Street Journal, Novartis said it "conducted an independent investigation [during the two months in question] with the support of external counsel to determine the extent of data discrepancies and whether they could be explained." It added that the internal probe had to be done in a "very confidential manner" to prevent senior officials at its AveXis unit from potentially interfering.
The probe formally launched in May included a "full technical quality investigation by our quality organisation…requiring significant time and resources, and the extensive review of physical and electronic documents, test data and laboratory notes," the company explained. In a report filed with the FDA about the data issue, Novartis said it began a formal investigation on May 8. The company plans to submit a full written explanation of the delay to the US regulator "as soon as this week," The Wall Street Journal reported.
Commenting on the news, the FDA stated that "while in some instances it may be reasonable for a firm to make a determination of the merit of a complaint in order to decide whether or not to initiate an investigation, each decision must be considered on a case-by-case basis." The agency added "we are not able to comment specifically on the circumstances of this case."
Earlier this month, Novartis disclosed that AveXis chief scientific officer Brian Kaspar and senior vice president of R&D Allan Kaspar had left the company in May. Although a source suggested that their departure was related to the data manipulation controversy, Brian Kasper denied any wrongdoing in the matter.
Meanwhile, US lawmakers have urged the FDA to pursue criminal, civil and regulatory action against Novartis concerning the alleged data alteration. The drugmaker also confirmed recently that it would respond to a request by the US Senate Finance Committee seeking details into the matter.
For related analysis, see ViewPoints: Novartis scrambles to cover its bases in data manipulation fallout. See also ViewPoints: Data manipulation an optical nightmare for Novartis.
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