Novartis Announces Data Confirming Benefit of Zolgensma in Pre-Symptomatic Children

23 June 2022

Novartis Announces Data Confirming Benefit of Zolgensma in Pre-Symptomatic Children

SPR1NT was a Phase III, multi-centre, single-arm study that investigated the efficacy and safety of onasemnogene abeparvovec (Zolgensma) in pre-symptomatic children with SMN1 mutations and either two or three copies of the SMN2 “back-up” gene. The participants were treated at 6 weeks of age or younger.

SPR1NT Trial participants were divided into 2 cohorts based on SMN2 copy number:

  • Cohort 1: 14 babies with two copies of SMN2 expected to develop SMA
     
  • Cohort 2: 15 babies with three copies of SMN2 expected to develop SMA
     

The results

Efficacy was compared with a matched natural-history cohort (23 babies).

Cohort 1:

  • All 14 enrolled infants sat independently for 30 seconds or more, at any visit ≤18 months
  • All survived without permanent ventilation at 14 months
  • 13 maintained body weight through 18 months
  • No child used nutritional or respiratory support
  • No serious adverse events were considered related to treatment by the investigator

Cohort 2:

  • All 15 children with three SMN2 copies treated before symptom onset, stood independently before 24 months (14 within the normal developmental window)
  • 14 walked independently (11 within the normal developmental window)
  • All survived without permanent ventilation at 14 months
  • 10 maintained body weight without feeding support through 24 months
  • None required nutritional or respiratory support
  • No serious adverse events were considered treatment-related by the investigator
     

Conclusion

Onasemnogene abeparvovec was effective for, and well-tolerated by, pre-symptomatic infants at risk of SMA Type 1 or Type 2, highlighting the urgency for early identification (newborn screening) and intervention.
 

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