NVS Novartis AG

By Thomas M. Burton 

This article is being republished as part of our daily reproduction of WSJ.com articles that also appeared in the U.S. print edition of The Wall Street Journal (August 23, 2019).

WASHINGTON -- Federal regulators examining test data manipulation for a gene-therapy drug made by Novartis AG are zeroing in on the company's two-month delay in launching a formal inquiry, according to documents and interviews.

The drug, Zolgensma, is used to treat a sometimes fatal form of spinal muscular atrophy in children and costs about $2.1 million for a one-time infusion. The Food and Drug Administration said this month that it wasn't informed about the data manipulation until after it approved use of the drug May 24.

FDA officials, who subsequently inspected the company's control testing lab in San Diego, noted that Novartis became aware of the problem March 14, but didn't open a formal probe until two months later, with no explanation for the gap.

"There is no documentation," FDA investigators Scott T. Ballard and Mihaly S. Ligmond wrote in their Aug. 2 report, as to why the probe "was not opened until 15 May, 2019, when the initial allegation is documented as having been reported on 14 March 2019."

In a statement to The Wall Street Journal, Novartis said that during those two months, it "conducted an independent investigation with the support of external counsel to determine the extent of data discrepancies and whether they could be explained."

Novartis said the internal probe had to be conducted in a "very confidential manner" so that senior executives at AveXis -- its Illinois-based unit that produced the drug -- wouldn't attempt to interfere. The company also said it plans to file a full written explanation of the gap to the FDA as early as this week.

In its statement, the company said that the investigation it formally launched in May was a "full technical quality investigation by our quality organization...requiring significant time and resources, and the extensive review of physical and electronic documents, test data and laboratory notes."

Novartis, in a report filed with the FDA about the data issue, said it began a formal investigation May 8.

Novartis announced last week that it had replaced two research executives at AveXis who led the research into the medicine, chief scientific officer Brian Kaspar and research chief Allan Kaspar. Allan Kaspar couldn't be reached. John Hueston, an attorney for his brother Brian Kaspar, said "he has cooperated in the internal investigation and categorically denies any wrongdoing."

In interviews, FDA officials said that two months to open an investigation seems excessive, especially when FDA action on a major drug was pending. They said the agency should have been notified as soon as the company suspected data manipulation, and that the data problems, had they been known, could have delayed approval.

The FDA, in an official statement Thursday, said, "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. We are not able to comment specifically on the circumstances of this case."

In a Twitter message Aug. 6, FDA Commissioner Ned Sharpless said agency officials "rely on truthful scientific data to make regulatory decisions, and we take the issue of data integrity very seriously." The FDA, he said, "will use its full authorities to take action, if appropriate, which may include civil or criminal penalties."

The data manipulation occurred in animal tests accompanying human clinical testing and involved giving various strengths of Zolgensma to mice to measure how long they lived. This was a small part of the overall testing.

Both the company and the federal agency said that the data manipulation, if known, wouldn't have affected the final decision on Zolgensma, which is considered the world's most expensive drug.

Write to Thomas M. Burton at tom.burton@wsj.com

(END) Dow Jones Newswires

August 23, 2019 02:47 ET (06:47 GMT)

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