AveXis Data Reinforce Effectiveness of Zolgensma™ in Treating Spinal Muscular Atrophy (SMA) Type 1

17 April 2019

Developed by AveXis, Zolgensma™ is a gene therapy product which works by replacing the missing or mutated SMN1 gene - the genetic cause of 5q SMA.

Zolgensma™ is currently in phase 3 clinical trials to test its safety and efficacy in infants with SMA Type 1 who are less than six months of age. The study was designed to enroll the broadest possible population of SMA Type 1 patients with one or two copies of the SMN2 backup gene and who have bi-allelic SMN1 gene deletion or point mutations.

The data shows:

  • Prolonged and event-free survival,
  • Early and rapid increases in CHOP-INTEND, which measures motor function in infants
  • Significant milestone achievement in SMA Type 1,
  • The new gene was inserted into the intended central nervous system targets, which led to widespread expression of SMN. This was comparable to results in tissue in the unaffected control group.
  • Of the 150+ patients treated with Zolgensma™, 5% of screened patients up to 5 years old were excluded due to an immune response against the virus.

This suggests that infants on Zolgensma™ live longer and achieve significant developmental milestones and the product is well tolerated. STR1VE is projected to complete in 2020.

Further information

On gene therapy
Novartis’ Press Release
STR1VE clinical trial