FDA approves Zolgensma gene therapy for infant SMA
The FDA has approved Zolgensma (onasemnogene abeparvovec-xioi) for patients under two years old with spinal muscular atrophy who carry bi-allelic mutations in the SMN1 gene. AveXis, a Novartis company, makes the drug. It is a one-time intravenous infusion that supplies a functional copy of the SMN gene, meant to sustain SMN protein expression and stop disease progression. Novartis says it is the first and only FDA-approved gene therapy for SMA, and it covers patients diagnosed before symptoms appear, not only those already showing signs of the disease.
This is an approved treatment, not an experimental one, so eligible infants can now get a one-time gene therapy for a disease that otherwise progresses fast and severely.
Source: AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA) (novartis.com).
Written by the Genomes desk from the primary source linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].