• Zolgensma is specifically designed to be given to children below the age of two years with SMA.
   
• The FDA acknowledged that there were data integrity issues with Zolgensma’s Biologics License Application.

The U.S. Food and Drug Administration, (FDA), recently announced a statement that there were data integration problems with Zolgensma’s Biologics License Applications (BLA). Meanwhile, the company stated that it is wholly confident about efficacy, quality, and safety of Zolgensma for treating spinal muscular atrophy (SMA) in children below the age of two years.

The FDA still supports the continued use and marketing of Zolgensma for patients with SMA. Novartis continues to be steadfast about this crucial treatment being readily available to pediatric patients with SMA. The company maintains complete evidence, that demonstrates effectiveness of the product and the safety profile, continues to provide convincing proof that backs the drug’s favorable benefit-risk profile.

Reports cite, that previously AveXis voluntarily disclosed to FDA and other authorities that some data presented earlier, as part of its BLA package, was inaccurate. Avexis became aware of data manipulation allegations in a specific animal testing process used during product development. The assessments in questions were taken for primary product testing and presently are not utilized for commercial product release.

This resulted in an immediate investigation to quickly find inferences and accordingly address the situation. Once Novartis had some interim conclusions, it shared its observations with FDA. As acknowledged by FDA, the data was a small portion of the company’s overall submission and was in an older process that is no more used.

According to Novartis, throughout the investigation, findings didn’t show any problems with quality, efficacy, or safety of the product. The company remains completely capable of facilitating fully compliant, high quality Zolgensma to patients.

Novartis has and will continue to work with FDA to update its submission and identify the quality gaps. Novartis is committed to make sure the greatest levels of integrity and transparency with health agencies and patients.

Source Credit:

https://www.novartis.com/news/media-releases/novartis-stands-behind-zolgensma-onasemnogene-abeparvovec-xioi-treatment-children-less-2-years-age-spinal-muscular-atrophy