- Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusion
- All children (100%) in the presymptomatic intravenous cohort of LT-002 maintained or achieved all assessed motor milestones, including independent walking
- To date, more than 3,000 children with spinal muscular atrophy have been treated with Zolgensma across clinical trials, managed access programs and in the commercial setting1
- Additionally, children with SMA Type 2 treated with investigational intrathecal OAV101 maintained or achieved new development gains
Novartis recently presented new data which underscore the transformational and sustained benefit of Zolgensma® (onasemnogene abeparvovec), an essential one-time gene therapy for the treatment of spinal muscular atrophy (SMA). 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 favorable. Highlighting the remarkable durability of Zolgensma, 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. During the time of LT-001, three additional patients also achieved the key milestone of “standing with assistance.”2
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.
Results from the IV cohort, which included 63 patients, demonstrated how a single administration of Zolgensma provided consistent, substantial and durable efficacy over time. Notably, 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.3 In total, six patients treated prior to 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 who were treated with one-time investigational OAV101 IT, were alive, free from permanent ventilation and continued to show incremental gains in motor function as of the May 2022 data cut-off. Five of 16 patients who had a milestone assessment achieved new milestones during the long-term follow-up period, such as crawling, walking or standing with assistance.3
The majority of patients in LT-002 (70.4%, 57/81) never received add-on therapy (76.2% of the IV cohort, 50% of the investigational OAV101 IT cohort). Among patients in the intravenous cohort, 24 of 25 (96%) patients treated before symptom onset achieved the motor milestone of walking alone prior to or without add-on therapy, and 30 of 32 (93.8%) patients treated after SMA symptom onset achieved the milestone of sitting without support prior to or without add-on therapy.
*Data presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.
Zolgensma® (onasemnogene abeparvovec) is the only approved gene therapy for the treatment of spinal muscular atrophy (SMA) and the only SMA treatment designed to directly address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.