Novartis Pledges More Transparency on Data Concerns

By Denise Roland 

Novartis AG, under fire for keeping a data-manipulation issue under wraps for several months, vowed to more quickly disclose any similar concerns in the future.

Chief Executive Vas Narasimhan on Monday told investors at an event in London that Novartis has committed to notify the Food and Drug Administration within five business days of any "credible allegation" of manipulation of data that had been submitted to the agency for drug approval.

The FDA last month said the Swiss company could face criminal penalties for holding back a data-manipulation concern with gene therapy medicine Zolgensma during its approval process. Novartis notified the FDA of the concern in late June, a month after the drug was approved, despite the issue being known inside the company since the middle of March.

Dr. Narasimhan has said the company wanted to investigate the specifics of the manipulation before bringing the concern to the agency. He said he was made aware of the issue in early May after an initial investigation found the allegations were credible.

But the timeline of those decisions has raised suspicions from the agency, and some politicians, that Novartis kept the issue under wraps because it didn't want to delay the approval of Zolgensma.

The medicine, which costs in the range of $2.1 million for a one-time infusion, treats children with an especially devastating, sometimes fatal form of spinal muscular atrophy. It is one of the first in a wave of gene therapies that promise to cure certain intractable diseases with one dose. They work by supplying a functioning version of the gene at fault.

The FDA gave Novartis the green light to sell Zolgensma in late May and the company put it on sale at a price of $2.1 million, making it the world's most expensive drug. Dr. Narasimhan has said the approval timeline for Zolgensma wasn't a factor.

"It's difficult for us to exercise judgment without it being later considered maybe not the best judgment, so we're just taking judgment out of the equation," Dr. Narasimhan said of the five-day pledge at the investor event, which focused environmental, social and corporate governance.

The FDA has said the data manipulation, which related to mouse studies carried out to compare different batches of Zolgensma, didn't affect its view that the gene therapy is safe and effective. It referred the matter to its Office for Criminal Investigations. Submitting false data to the agency as part of a new-drug application could be a crime if investigators prove the actions were intentional and not an oversight.

The episode has also drawn criticism from several high-ranking politicians including Democratic presidential candidates Sens. Elizabeth Warren of Massachusetts and Bernie Sanders of Vermont, and Sen. Chuck Grassley (R., Iowa).

Write to Denise Roland at [e-mail]

(END) Dow Jones Newswires

September 09, 2019 12:38 ET (16:38 GMT)

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