SMA type 1: the results of the STR1VE-EU trial confirm the efficacy of Zolgensma

The STR1VE-EU trial evaluated the safety and efficacy of a single intravenous infusion of Zolgensma (onasemnogene abeparvovec) in 33 infants with SMN1-related proximal spinal muscular atrophy (SMA) type 1, presenting one or two copies of the SMN2 gene and aged under 6 months.

The results of this trial, published in October 2021, show that:

  • 97% of participants do not require permanent ventilatory support at 14 months of age, versus 26% in the natural history cohort,
  • 44% are able to sit independently for at least 10 seconds at 18 months of age, versus 0% in the same natural history cohort,
  • Zolgensma is well tolerated. The most commonly reported adverse effects include fever, upper respiratory tract infections and elevated transaminase levels.

These data confirm the significant improvement obtained during the STR1VE trial, an American trial with the same design, conducted among 22 participants, and the results of which appeared in April 2021.

 

STR1VE-EU study group. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Mercuri E, Muntoni F, Baranello G, et al. Lancet Neurol., 2021 (Oct). 20(10):832-841.   Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. J W Day, R S Finkel, C A Chiriboga, et al. Lancet Neurol. 2021 (Avr).20(4):284-293.