Better known risks of Zolgensma

Zolgensma® (onasemnogene abeparvovec) is the first gene therapy product to obtain marketing authorization in 2019 in a neuromuscular disease, namely SMA. 

Various serious side effects have been reported with this product: hepatotoxicity, transient thrombocytopenia, cardiac disorders, thrombotic microangiopathy… 

An exhaustive analysis of data from preclinical studies, clinical trials (around 100 patients with SMA in total) or post-marketing (665 patients) shows that these risks are rare and can be anticipated or managed:

  • Taking prednisolone helps reduce hepatotoxicity, 
  • The drop in platelet count is transient,
  • Thrombotic microangiopathy may require medical intervention.

 

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Day JW, Mendell JR, Mercuri E et al. Drug Saf. 2021 (Oct). 44(10):1109-1119.