Gene therapy for paediatric patients with SMA in US

August 6, 2019 0 By FM

AveXis, a Novartis company, announced the US FDA has approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of paediatric 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. It is the first gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.

The approval of Zolgensma is based on data from the ongoing Phase 3 STR1VE trial and the completed Phase 1 START trial evaluating the efficacy and safety of a one-time IV infusion of Zolgensma in patients with SMA Type 1 who showed symptoms of SMA at <6 months of age, with one or two copies in the STR1VE trial or two copies in the START trial of the SMN2 backup gene and who have bi-allelic SMN1 gene deletion or point mutations.

These data show Zolgensma provides unprecedented rates of survival never seen in the natural history of the disease; rapid motor function improvement, often within one month of dosing; and, durable milestone achievement, including the ability to sit without support, a milestone never achieved in untreated patients.