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Novartis Defends $2.1 Million Gene Therapy After FDA Data Manipulation Claims—Brainstorm Health

Good afternoon, readers.

Shares of Novartis fell nearly 3% in Tuesday trading after the Food and Drug Administration (FDA) levied serious allegations of data manipulation against the drug giant. The claims involve the gene therapy Zolgensma, used to treat the devastating childhood rare disorder spinal muscular atrophy (SMA) - and which, at a $2.1 million list price, is the most expensive drug in the world.

Novartis purchased AveXis, the company that manufactures Zolgensma, in 2018, and received FDA approval for the drug last year. The FDA said on Tuesday that, "following the FDA’s approval of the product, the agency was informed by AveXis Inc., the product’s manufacturer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA."

The agency didn't say that these manipulations would warrant yanking Zolgensma's approval. In fact, it said that it is "carefully assessing this situation and remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application."

Still, acting FDA Commissioner Ned Sharpless shot a clear warning across Novartis' bow. That includes the specter of potential criminal charges. "We rely on truthful scientific data to make regulatory decisions, and we take the issue of data integrity very seriously. In this case, the agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties," Sharpless wrote in a tweet.

Novartis defended the integrity of the Zolgensma application, noting that the data issues were self-reported.

"At no time during the investigation did the findings indicate issues with product safety, efficacy, or quality. We remain fully capable of releasing high-quality, fully compliant Zolgensma to patients in need. We have and will continue to work in close cooperation with the FDA to appropriately update our submission and address any quality gaps identified," the company wrote in a statement.

Read on for the day's news.

Sy Mukherjee, @the_sy_guy, sayak.mukherjee@fortune.com

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