Novartis gets FDA OK for 1st gene therapy for spinal muscular atrophy

• SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2[1],[2]
• Zolgensma (onasemnogene abeparvovec-xioi) is approved for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) including those who are pre-symptomatic at diagnosis
• Zolgensma is designed to address the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion
• Data from the Phase 3 STR1VE trial show prolonged event-free survival, increases in motor function and significant milestone achievement in patients with SMA Type 1, consistent with the Phase 1 START trial
• In the START trial, patients treated with Zolgensma achieved motor milestones never seen in the natural history of the disease, including sitting, talking and some patients walking, with no waning of effect nearly four years post-dosing

AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma^® (onasemnogene abeparvovac-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.

http://www.globenewswire.com/news-release/2019/05/24/1849953/0/en/AveXis-receives-FDA-approval-for-Zolgensma-the-first-and-only-gene-therapy-for-pediatric-patients-with-spinal-muscular-atrophy-SMA.html