AveXis, a pharmaceutical company owned by Novartis, has obtained the European Commission (EC) conditional approval for its Zolgensma (onasemnogene abeparvovec) to treat a type of spinal muscular atrophy (SMA).

The EC approval indicated Zolgensma for SMA patients with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene, and covers children with SMA, weighing up to 21kg.

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.

“Additionally, we have met with more than 100 stakeholder organizations across Europe to discuss our “Day One” access program to enable rapid access with customisable options designed to work within local pricing and reimbursement frameworks.”

Phase 3 STR1VE-US and Phase 1 START trials evaluated Zolgensma

Zolgensma is a one-time gene therapy intended for progressive genetic disease. The drug is consistently priced under a value-based framework, however final pricing and reimbursement decisions are finalised at the local level.

The company has designed the “Day One” access program to work within existing, local pricing and reimbursement frameworks, and provide health and reimbursement bodies with flexible options to support quick access and broad reimbursement.

The regulatory approval is supported by the Phase 3 STR1VE-US and Phase 1 START trials that evaluated the efficacy and safety of Zolgensma in symptomatic SMA Type 1 patient, who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene.

In STR1VE-EU, a comparable ongoing Phase 3 clinical trial, Zolgensma showed prolonged event-free survival; rapid motor function improvement and, achieved certain sustained milestones including the ability to sit without support, crawl and walk independently.

In addition, interim results from the ongoing SPR1NT trial, a Phase 3, open-label, single-arm study of Zolgensma has also supported the approval.