Novartis subsidiary AveXis has received conditional approval from the European Commission (EC) for Zolgensma (onasemnogene abeparvovec) to treat spinal muscular atrophy (SMA).

The approval is for infants and young children with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1 or 5q SMA with a bi-allelic mutation in the SMN1 gene, as well as up to three copies of the SMN2 gene.

Zolgensma is a one-time gene therapy intended to target the genetic root cause of the disease via replacement of the function of the missing or nonworking SMN1 gene.

Given as a single, intravenous infusion, the drug delivers a new working copy of the SMN1 gene into a patient’s cells, stopping disease progression.

AveXis president Dave Lennon said: “The EC approval of Zolgensma is a significant milestone for the SMA community, and further underscores the substantial clinical value of the only gene therapy for SMA, bringing new hope to those impacted by this rare, but devastating disease.

“Even under the current pandemic conditions, the urgent need to treat SMA has resulted in access pathways in France and Germany for Zolgensma, a potentially life-saving medicine delivered in a single dose.”

EC approval is supported by data from Phase III STR1VE-US and Phase I START clinical trials that assessed the safety and efficacy of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patient.

Participants were aged under six months at dosing with one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. A comparable Phase III study called STR1VE-EU is ongoing.

More supportive data included interim findings from the ongoing SPR1NT trial, a Phase III  a single, one-time IV infusion of the drug in pre-symptomatic patients with bi-allelic deletion of SMN1 with two or three copies of SMN2.

AveXis also activated ‘Day One’ access that provides programme for ministries of health and reimbursement bodies with different options for access and reimbursement.

In March, the Japanese Ministry of Health, Labour and Welfare (MHLW) approved Zolgensma to treat patients with SMA.