Novartis stands behind Zolgensma for the treatment of children less than 2 years of age with spinal muscular atrophy.

The FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma (onasemnogene abeparvovec-xioi). First and foremost, Novartis are fully confident in the safety, quality and efficacy of Zolgensma. The FDA supports the continued marketing and use of Zolgensma for patients with spinal muscular atrophy (SMA) less than 2 years of age.

On June 28th, AveXis voluntarily self-disclosed to the FDA and subsequently to other health authorities that some data previously submitted to the agency as part of the BLA package was inaccurate. AveXis had become aware of allegations of data manipulation in a specific animal testing procedure used in the development of the product. The assays in question were used for initial product testing and are not currently used for commercial product release. An investigation was immediately initiated to rapidly understand any implications and address the situation. Once Novartis had interim conclusions from its investigations, it shared its findings with the FDA. As noted by the FDA, the data in question were a small portion of the overall submission and are limited to an older process no longer in use.At no time during the investigation did the findings indicate issues with product safety, efficacy or quality.

AveXis /Novartis maintains that the totality of the evidence demonstrating the product’s effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile. The companies remain steadfast that this important treatment remain available to pediatric patients with SMA less than 2 years of age.

Novartis/AveXis remains fully capable of releasing high-quality, fully compliant Zolgensma to patients in need. The companies have and will continue to work in close cooperation with the FDA to appropriately update our submission and address any quality gaps identified. The companies are committed to ensuring the highest levels of transparency and integrity with health agencies, as well as with the patients and providers we serve.

We do not expect this to impact the timing of our ongoing Zolgensma regulatory filings and development programs. AveXis is committed to taking appropriate action to prevent future incidents across its portfolio of development programs.