Novartis Reveals Long-Term Data Of Atrophy Gene Therapy Exhibiting Sustained Durability Of Up To 7.5 Years

Novartis AG NVS has presented new data on Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA).
The latest data from two Long-Term Follow-Up studies, LT-001 and LT-002, show the continued efficacy and durability of Zolgensma across a range of patient populations, with an overall benefit-risk profile that remains favorable.
Highlighting the remarkable durability of Zolgensma, data from LT-001, an ongoing 15-year study of patients who completed the Phase 1 START study, showed that up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.
During LT-001, three additional patients also achieved the key milestone of “standing with assistance.”
Interim results from the 15-year LT-002 study, which includes both presymptomatic and symptomatic patient populations, were also presented. All patients (100%) maintained motor milestones achieved during their respective parent studies in the follow-up period.
Results from the IV cohort, which included 63 patients, demonstrated how a single administration of Zolgensma provided consistent, substantial, and durable efficacy over time.
In the presymptomatic IV cohort (n=25), all children (100%) either maintained the highest milestone achieved during the parent study (walking alone) or achieved the milestone by the data-cut off.
Six patients treated before SMA symptom onset and 16 treated after SMA symptom onset achieved new motor milestones in the follow-up period.
All 18 children in LT-002 treated with one-time investigational OAV101 IT were alive, free from permanent ventilation, and showed incremental gains in motor function as of the May 2022 data cut-off.
Five of 16 patients with a milestone assessment achieved new milestones during the long-term follow-up period, such as crawling, walking, or standing with assistance.
Price Action: NVS shares are up 0.72% at $82.78 during the premarket session on the last check Monday.

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