This approval makes onasemnogene abeparvovec the only gene therapy available in the EU for the treatment of SMA.48

Onasemnogene abeparvovec is indicated for the treatment of patients with 5q SMA with a biallelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a biallelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.49

It delivers a fully functioning copy of the human SMN1 gene to promote the survival and function of motor neurons.48,49