Novartis' gene therapy arm AveXis has announced European approval for Zolgensma (onasemnogene abeparvovec), the only gene therapy for spinal muscular atrophy (SMA).

Specifically, the European Commission has granted conditional 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 for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.

The clearance covers babies and young children with SMA up to 21kg according to the approved dosing guidance.

In Europe each year, approximately 550-600 infants are born with SMA, a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, which causes rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.

Zolgensma is a one-time gene therapy administered via an intravenous infusion which is designed to address the genetic root cause of the disease by replacing the function of the missing or nonworking SMN1 gene, halting disease progression.

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, said Dave Lennon, president of AveXis.

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 programme to enable rapid access with customisable options designed to work within local pricing and reimbursement frameworks.

Designed to work within existing, local pricing and reimbursement frameworks, the 'Day One' access programme offers ministries of health and reimbursement bodies a variety of flexible options that can be implemented immediately to support swift access and broad reimbursement.

It ensures the cost of patients treated before national pricing and reimbursement agreements are in place align with the value-based prices negotiated following clinical and economic assessments.

AveXis says the programme offers a variety of customisable options including:

The EC approval is based on the Phase IIISTR1VE-US and Phase I START trials that evaluated the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients <6 months of age at dosing, who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. STR1VE-EU, a comparable Phase III study is ongoing.

According to AveXis, Zolgensma showed prolonged event-free survival; rapid motor function improvement, often within one month of dosing; and, sustained milestone achievement, including the ability to sit without support, crawl and walk independently milestones never achieved in untreated Type 1 patients.

The approval of Zolgensma represents an important new way for physicians to treat patients with SMA, said Dr Eugenio Mercuri, Professor, Pediatric Neurology, Catholic University, Rome, Italy. The results we have seen for Zolgensma to date from the STR1VE clinical trial show an impressive survival rate at the conclusion of the study, with the majority of patients achieving functional milestones, like sitting without support, that wouldn't have been reached in untreated infants.

AveXis said it intends to make onasemnogene abeparvovec available across the UK as soon as possible so patients and their families can benefit from the treatment.

Evaluation of onasemnogene abeparvovec by NICE to determine NHS reimbursement is already underway, and "we will continue to work closely with relevant organisations across the UK to help them complete their assessments," the firms added, also noted that it had already engaged with key UK stakeholders including patient advocacy groups and clinicians.

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EU approves Novartis' SMA gene therapy Zolgensma - PharmaTimes