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Novartis says former AveXis execs hindered internal probe into Zolgensma data

Novartis AG said an internal investigation into manipulation of preclinical data on gene therapy Zolgensma was "drawn out" by two now-former executives of its unit AveXis Inc.

The Swiss drugmaker acquired AveXis in April 2018 in an $8.7 billion deal. AveXis developed spinal muscular atrophy treatment Zolgensma, approved in May and priced $2.1 million — the most expensive drug on the U.S. market.

In August, the U.S. Food and Drug Administration opened a probe into Zolgensma after AveXis reported a data manipulation issue related to animal testing that was done to support the therapy's approval. Just over a week after the investigation was launched, Novartis said AveXis' Head of Research and Development Allan Kaspar and Chief Scientific Officer Brian Kaspar were no longer with the company.

In its response letter to the FDA, Novartis said an employee of AveXis alleged that two AveXis senior executives "altered or instructed others to alter" certain data related to the preclinical testing — which led to the launch of the internal probe on March 14.

The letter, dated Aug. 23, was released by the FDA on Sept. 24, Reuters reported.

"Because the allegations involved two founders who were still senior executives and fixtures at AveXis, and because the allegations involved data manipulation, this initial internal investigation was led by external counsel to ensure that key evidence was preserved, efficiently gathered, and made available for a thorough technical review without interference from the two implicated senior AveXis executives," Novartis said in the letter.

The Swiss drugmaker added that the internal probe was "drawn out" due to the two former executives' "lack of cooperation" and "categorical denial" of the data manipulation allegations.

U.S. FDA probe

Novartis previously said it knew about inaccurate data on Zolgensma as early as mid-March while the U.S. FDA was reviewing the drug for approval but did not inform the regulator until after months later. The drugmaker's CEO Vas Narasimhan, in an Aug. 7 call said the data inaccuracies stemmed from an older, discontinued test and they the inaccuracies would not affect the ultimate drug's efficacy, safety and product quality.

The company "proactively" informed the FDA of its findings June 28, leading to the FDA's onsite inspections in early August. The regulator then stated that "civil or criminal penalties" may be appropriate.

However, the agency said the data issues do not impact the drug's benefits shown during clinical trials.

"These data do not change the agency's positive assessment of the information from the human clinical trials that were conducted as part of the development program," the agency said Aug. 6. The FDA "remains confident that Zolgensma should remain on the market."

On Sept. 19, Novartis said a final autopsy report showed that the death of an infant in a phase 3 study was unrelated to Zolgensma. AveXis said the death of a child in the late-stage trial, called STR1VE-EU, was caused by hypoxic-ischemic brain damage, with respiratory tract infection being the underlying cause.