Novartis's Data Reinforces Age-Appropriate Development With Zolgensma Gene Therapy Study

Novartis AG NVS announced new data from the completed Phase 3 SPR1NT study of Zolgensma (onasemnogene abeparvovec), a one-time treatment for spinal muscular atrophy (SMA).
The data demonstrated that children with three copies of the SMN2 backup gene who were treated presymptomatically achieved age-appropriate motor milestones, including standing and walking.
In addition, a descriptive posthoc analysis of START, STR1VE-EU, and STR1VE-US (n=65) indicated children with SMA Type 1 achieved or maintained necessary measures of bulbar function, including the ability to speak; swallow, and meet nutritional needs; and maintain airway protection.
Fourteen patients (93%) walked independently, 11 of whom achieved this milestone within the WHO window of normal development.
All patients were independent of nutritional and respiratory support for the study duration.
All patients experienced at least one adverse event (AE) after dosing, eight (53%) of which were considered treatment-related. There were no serious, treatment-related AEs.
Three patients were reported to have had severe adverse events (SAEs), all of which resolved and were not related to treatment.
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