In May, the FDA approved Novartis’ gene therapy Zolgensma as a one-time treatment for spinal muscular atrophy. Recently, the regulatory agency revealed that data manipulation was involved in the preclinical process but suggested that the therapy remain on the market.
On June 28, more than a month after the agency approved the gene therapy, AveXis Inc., a Novartis subsidiary, informed the FDA “about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application.”
Novartis has said it stands behind the Zolgensma data. According to the company, the assays in question were used for initial product testing and are not currently used for commercial product release.
The FDA remains confident about the efficacy and safety of Zolgensma; however, the integrity of the product-testing data used in the development of the product’s manufacturing process is still being evaluated. AveXis became aware of the data manipulation issue prior to the FDA’s approval of the therapy. The FDA said it will “use its full authorities to take action” if necessary. That could include civil or criminal penalties.
Novartis said it does not anticipate the issues regarding the preclinical data manipulation to impact the timing of ongoing Zolgensma regulatory filings.
Source: BioSpace, August 7, 2019