The data set in the Full Prescribing Information includes 44 patients who were treated with ZOLGENSMA and ranged in age from 0.3 to 7.9 months at the time of infusion.¹

Adverse reactions and ALT increases (laboratory finding) following treatment with ZOLGENSMA (n=44)

ALT=alanine aminotransferase; ULN=upper limit of normal.
^aAE data come from the ZOLGENSMA Prescribing Information. This is based on September 27, 2018 data cut off. 
^b41 patients received the therapeutic or higher dose of ZOLGENSMA, and 3 patients received a lower dose.
^cElevated aminotransferases include alanine aminotransferase and/or aspartate aminotransferase.
^dOne patient (the first patient enrolled in NCT02122952) was enrolled prior to the protocol amendment instituting administration of prednisolone pre- and post-ZOLGENSMA infusion.

Acute serious liver injury, acute liver failure, and elevated aminotransferases can occur with ZOLGENSMA¹

Acute serious liver injury, acute liver failure, and elevated aminotransferases can occur with ZOLGENSMA. Hepatotoxicity (which may be immune-mediated), generally manifested as elevated ALT and/or AST levels. Acute serious liver injury and acute liver failure, including fatal cases, have been reported with ZOLGENSMA use. In order to mitigate potential aminotransferase elevations, administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion.¹

• Immune-mediated hepatotoxicity may require adjustment of the corticosteroid treatment regimen, including longer duration, increased dose, or prolongation of the corticosteroid taper

Patients with preexisting liver impairment or acute hepatic viral infection may be at higher risk of acute serious liver injury/acute liver failure. Patients with ALT, AST, or total bilirubin levels (except due to neonatal jaundice) > 2 × ULN have not been studied in clinical trials with ZOLGENSMA. Carefully consider the risks and benefits of ZOLGENSMA therapy in patients with preexisting liver impairment.¹

Although in the clinical trials and in postmarketing experience, asymptomatic aminotransferase elevations were very commonly reported, in the managed access program and in the postmarketing setting, cases of acute serious liver injury and acute liver failure, including a few cases with fatal outcomes, have been reported.¹

• Some patients have experienced elevations in ALT and AST > 20 × ULN, prolonged prothrombin time and have been symptomatic (eg, vomiting, jaundice), which required the use of corticosteroids, sometimes with prolonged duration and/or a higher dose

• If acute serious liver injury or acute liver failure is suspected, consult a pediatric gastroenterologist or hepatologist

In order to help manage a possible increase in liver aminotransferases, all patients should be treated with systemic corticosteroids before and after infusion. See Full Prescribing Information for important treatment steps, including corticosteroid recommendations, laboratory testing, and monitoring.¹

The following adverse reactions have been identified during post-approval use of ZOLGENSMA¹:

• Thrombotic microangiopathy and thrombocytopenia

• Acute liver failure (fatal and non-fatal) and acute liver injury

• Pyrexia

• Troponin increased

Because these reactions are reported voluntarily, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.