SMA Newsroom

May 4, 2023

New interim data on long-term efficacy and safety data up to 7.5 years post-dosing of Zolgensma.

Novartis announced new long-term data for Zolgensma® (onasemnogene abeparvovec).

The latest data from two Long-Term Follow-Up (LTFU) 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 favourable, with treated children maintaining motor milestones up to 7.5 years post-dosing.

Data from LT-001, an ongoing 15-year LTFU 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.

Interim results from the 15-year LT-002 study, which includes both presymptomatic and symptomatic patient populations, as well as intravenous (IV) and intrathecal (IT) administration methods were also presented, with all patients (100%) maintaining motor milestones achieved during their respective parent studies in the follow-up period.

The data were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March 2023.