Zolgensma® receives conditional approval for spinal muscular atrophy in Europe

AveXis (Basel, Switzerland) has announced that their gene therapy product, Zolgensma® (onasemnogene abeparvovec), has received conditional approval by the European Commission for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1, or SMA patients with up to three copies of the SMN2 gene. Zolgensma is administered as a one-time intravenous gene therapy and was designed to address the genetic root cause of SMA by replacing the function of the missing, or nonworking, SMN1 gene. It works by delivering a new working copy of the SMN1 gene into a patient’s cells and halting...