Posted by Michael Wonder on 19 May 2020
AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy
19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe 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.
AveXis today announced the European Commission granted conditional approval for 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.
The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.
AveXis in discussions with EU governments and reimbursement agencies to agree on terms of innovative “Day One” access program to enable rapid access in all EU countries given urgent need to treat SMA.
