Zolgensma Labeling Updated With Additional Safety Information

Zolgensma is an adeno-associated virus vector-based gene therapy.

The Food and Drug Administration (FDA) has approved several revisions to the prescribing information for Zolgensma (onasemnogene abeparvovec-xioi). 

Zolgensma is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron (SMN1) gene.

The Boxed Warning has been updated with the addition of acute liver failure, which can occur with Zolgensma use. Cases of acute serious liver injury, acute liver failure, and elevated aminotransferases have been reported with Zolgensma. The risks and benefits of Zolgensma should be carefully weighed in patients with preexisting liver impairment. A pediatric gastroenterologist or hepatologist should be consulted if liver function abnormalities continue to persist at levels at least 2 x ULN after the 30-day period of systemic corticosteroids.

Health care professionals are advised to administer systemic corticosteroids to all patients before and after Zolgensma infusion in order to avoid potential aminotransferase elevations. Intravenous corticosteroids can be considered if oral corticosteroid therapy is not tolerated.

Additionally, due to the increased risk of serious systemic immune response, Zolgensma treatment should be postponed in patients with concurrent infections until the infection has resolved. Clinical signs or symptoms of infection should not be evident at the time of Zolgensma administration.


Zolgensma. Package insert. Novartis Gene Therapies, Inc.; 2021. Accessed October 27, 2021. https://www.fda.gov/media/126109/download.