In Wake of Zolgensma Scandal, Novartis Pledges More Sunshine on Data Integrity

Clinical Trial Data

Novartis Chief Executive Officer Vas Narasimhan has pledged the company will be more forthcoming with the U.S. Food and Drug Administration and other regulatory agencies regarding data mismanagement and manipulations in the future.

On Monday, Narasimhan spoke with investors regarding corporate governance in the wake of the Zolgenzma data manipulation scandal that has plagued the Swiss pharma giant for the past month. Shares of Novartis are slumping in premarket trading after reports confirmed that the company knew of discrepancies in the data surrounding its gene therapy treatment Zolgensma and withheld the information.

Narasimhan confirmed the company knew of the data manipulation that occurred in the early development of the gene therapy treatment for spinal muscular atrophy, but withheld informing the U.S. Food and Drug Administration until after the treatment had been approved for use in the United States. Zolgensma was approved by the FDA in May, but Novartis alerted the regulatory agency to concerns over data manipulation in June. In August, the FDA revealed the manipulation but opted to keep the gene therapy on the market. The FDA said the data manipulation was “limited to only a small portion of the product testing data that was contained in the marketing application.”

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Novartis only informed the FDA after completing its own internal investigation, the company said. Novartis acquired AveXis last year for $8.7 billion. The parent company has stood behind the data submitted to the FDA. In its support of the gene therapy, Novartis said the assays that were in question regarding the data manipulation were used for initial product testing but are not currently used for commercial product release.

With that delay in informing the regulatory agency, Novartis could be facing civil and criminal penalties, CNBC reported Monday. With this backdrop in mind, Narasimhan pledged to be more forthcoming about future discrepancies, should they occur. During a call with investors, Narasimhan said the company is committed to notifying the FDA within five business days of any allegations regarding data integrity that could impact any pending applications, CNBC reported.

“What we realized through this situation is, during a filing, it’s difficult for us to exercise that judgment without it being later considered maybe not the best judgment,” Narasimhan said, according to CNBC. “So we’re just taking judgment out of that equation,” with the new five-day rule.

Narasimhan told the investors that his company gave “detailed explanations” regarding the internal investigation into the manipulation and explained why the company waited before it disclosed the issue.

Following the revelation of the manipulation, Novartis terminated some of the researchers involved with the development of Zolgensma at its subsidiary, AveXis. Last month, the Illinois-based company said Brian and Allen Kaspar have not been involved in any operations at AveXis since early May.

Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis. The gene therapy was approved on the basis of ongoing data from the Phase III STRIVE trial that evaluated the efficacy and safety of a one-time IV infusion of Zolgensma in patients with SMA Type 1 who showed symptoms of SMA at less than six months of age.

The revelation of the data manipulation, plus the $2.1 million list price tag for Zolgensma, has raised the ire of multiple federal lawmakers, including Democratic presidential candidates Sens. Bernie Sanders and Elizabeth Warren. The senators, along with other colleagues from the upper chamber of Congress, issued a letter to the FDA demanding that the regulatory agency take action against AveXis.

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