New Data Shows Long-Term Benefits of Zolgensma in SMA Patients

BioSpace | September 20, 2019

While the approval of AveXis’ gene therapy for spinal muscular atrophy has been tainted by the revelation of early data manipulation, the Novartis subsidiary presented some long-term data at a conference that backs up the treatment’s efficacy. On Thursday, AveXis presented new data from two Phase III trials, the SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase III STR1VE clinical program for Zolgensma that showed positive outcomes demonstrating age appropriate major milestone gain with pre symptomatic treatment and prolonged event-free survival in patients with SMA Type 1. Data from these two trials, as well as long-term follow-up data to the Phase I START study will be presented at the 2019 European Pediatric Neurology Society (EPNS) Congress. Updated results from the STR1VE study demonstrate that the gene therapy Zolgensma(onasemnogene abeparvovec-xioi)has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients. AveXis said. Interim data from the SPRINT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age appropriate major milestone gain, the company added in its announcement. Olga Santiago, AveXis’ chief medical officer, said the data being presented at EPNS “demonstrate the life-changing impact Zolgensma can have on children with SMA Type 1.” “It is critical to diagnose SMA and begin treatment as early as possible in order to stop irreversible motor neuron loss and make the achievement of major motor milestones such as crawling, sitting and walking a possibility,” Santiago said in a statement.

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