Zolgensma™

What is Zolgensma™?

Zolgensma™ (onasemnogene abeparvovec-xioi) is the second treatment approved in Europe to treat SMA in babies and young children.

At a glance

Zolgensma™ facts

Zolgensma™ is as gene therapy, which means it works on the genetic root cause of SMA: it provides a new functional copy of the SMN1 gene, which is faulty or missing in people living with SMA. It is administered as a one-time, intravenous (IV) infusion.

  • Zolgensma received conditional marketing authorisation in the European Union in May 2020. Following the collection of additional data and post-marketing requirements, Zolgensma received full approval in May 2022.
  • Zolgensma was initially developed by AveXis, acquired by Novartis in 2018 and renamed Novartis Gene Therapies.

Specification

The European Commission granted full 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 

  • 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. 

Further Reading

Zolgensma™ Resources