Posted by Michael Wonder on 28 Mar 2020
AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy
28 March 2020 - AveXis, a Novartis company, today announced that the CHMP of the EMA has adopted a positive opinion recommending conditional marketing authorisation of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (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.
Zolgensma is administered during a single intravenous infusion, delivering a new working copy of the SMN gene into a patient’s cells, halting disease progression.
The European Commission reviews the CHMP recommendation and usually delivers its final decision in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway, Liechtenstein and the United Kingdom.
