- Novartis AG (NYSE: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.
- Price Action: NVS shares are up 2.32% at $84.28 during the market session on the last check Monday.
