The US FDA remains confident that Zolgensma will not be pulled after being alerted to allegations of data manipulation by the gene therapy manufacturer AveXis.

In May 2019, Novartis company AveXis received US Food and Drug Administration (FDA) approval for its $2.1 million single-dose, one-time gene therapy Zolgensma (onasemnogene abeparvovec) to treat children less than two years of age with spinal muscular atrophy (SMA).

A month later, AveXis informed the Agency “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),” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER) wrote in a statement yesterday.

Image: AdobeStock/Elnur

“The FDA 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.”

But while Zolgensma may not be pulled, Marks said AveXis could be subject to civil or criminal penalties because the firm became aware of the data manipulation issue and did not inform the FDA before the gene therapy won approval.

A statement issued by Novartis said AveXis immediately undertook an investigation to understand any implications and address the situation once the data issues came to light.

“Once we had interim conclusions from our investigations, we shared our findings with the FDA. As noted by the FDA, the data in question were a small portion of our overall submission and are limited to an older process no longer in use.”

The firm said: “At no time during the investigation did the findings indicate issues with product safety, efficacy or quality,” adding that it does not expect this to impact ongoing Zolgensma regulatory filings and development programs.

“AveXis is committed to taking appropriate action to prevent future incidents across its portfolio of development programs.”

Related news: ‘Zolgensma: We all know the price, but how is it made?’