Posted by Michael Wonder on 06 Aug 2019
Novartis stands behind Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy
6 August 2019 - Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma (onasemnogene abeparvovec-xioi). First and foremost, we 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.
We maintain that the totality of the evidence demonstrating the product’s effectiveness and its safety profile continue to provide compelling evidence supporting an overall favourable benefit-risk profile. We remain steadfast that this important treatment remain available to paediatric patients with SMA less than 2 years of age.
