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.