What is Zolgensma™?
Zolgensma™ (onasemnogene abeparvovec-xioi) is the second treatment approved to treat SMA in Europe in babies and young children.
Zolgensma™, referred to as gene therapy, is administered during a single, intravenous (IV) infusion that is designed to address the genetic root cause of SMA by replacing the function of the missing or faulty SMN1 gene.
At a glance
- Zolgensma is (conditionally) approved in many European countries for the treatment of babies and young children (see specifications above).
- Zolgensma is given through an intravenous (IV) infusion. It is a one-time treatment.
- Zolgensma is a “gene therapy” (replacement of the function of the missing or non-working SMN1 gene).
- Zolgensma was developed by AveXis and then by Novartis Gene Therapies.
The European Commission granted conditional approval for Zolgensma™ 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.
The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.