AveXis (Basel, Switzerland) has announced that their gene therapy product, Zolgensma® (onasemnogene abeparvovec), has received conditional approval by the European Commission 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.
Zolgensma is administered as a one-time intravenous gene therapy and was designed to address the genetic root cause of SMA by replacing the function of the missing, or nonworking, SMN1 gene. It works by delivering a new working copy of the SMN1 gene into a patient’s cells and halting disease progression.
“The European Commission 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,” commented Dave Lennon, President of AveXis.
“…we have met with more than 100 stakeholder organizations across Europe to discuss our ‘Day One’ access program to enable rapid access with customizable options designed to work within local pricing and reimbursement frameworks,” Lennon added.
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This conditional approval is based on data from two clinical trials – the Phase III STR1VE-US trial and the Phase I START trial – which evaluated the safety and efficacy of one-time intravenous 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.
A comparable Phase III trial, known as STR1VE-EU, is currently ongoing.
Zolgensma demonstrated prolonged event-free survival, rapid motor function improvement (often within 1 month of dosing) and sustained milestone achievements, including the ability to sit without support, crawl and walk independently. These milestones have never been achieved in untreated Type 1 patients.
“The approval of Zolgensma represents an important new way for physicians to treat patients with SMA. 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,” concluded Eugenio Mercuri (Catholic University, Rome, Italy).