Novartis Gene Therapies unveils new interim data from ongoing Phase III STR1VE-EU trial of Zolgensma (onasemnogene abeparvovec) in patients with spinal muscular atrophy (SMA) Type 1

Data showed 65.6% patients on Zolgensma achieved motor milestones not observed in natural history of SMA Type 1, including:18.8% who could sit independently for ≥10 seconds (primary efficacy endpoint); 66.7% who gained head control and 25% who were able to roll from back to sides

SPS commentary:

The European Commission granted conditional approval for Zolgensma in May 2020 for the treatment of patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.

PharmaTimes