AveXis receives European Commission (EC) approval for the gene therapy Zolgensma®
19 May 2020
The EC has granted approval for Zolgensma® (onasemnogene abeparvovec) for the treatment of babies and young children who have 5q SMA with a bi-allelic mutation in the SMN1 gene and either a clinical diagnosis of SMA Type 1 or up to three copies of the SMN2 gene.
According to the approved dosing guidance this covers babies and young children who have SMA up to 21 kg. AveXis UK will be updating the UK SMA Community as soon as possible with what this means for potential access in the UK.