Zolgensma Long-Term Data Demonstrates Sustained Durability for Spinal Muscular Atrophy


Children in the LT-001 study treated after onset achieved or maintained additional milestones up to 7.5 years after 1-time intravenous infusion, Novartis says.

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The latest data from 2 long-term follow up studies, LT-001 (NCT03421977) and LT-002 (NCT04042025), demonstrated the continued durability and efficacy of onasemnogene abeparvovec (Zolgensma) from Novartis), a 1-time gene therapy for the treatment of spinal muscular atrophy (SMA), which also had an overall benefit-risk profile that remains favorable.

“Data from the LT-001 and LT-002 studies showed that, regardless of the patient’s symptomatic status at the time of treatment, [onasemnogene abeparvovec] [intravenous (IV)] is an effective and durable treatment option. As the number of patients treated with gene therapy around the world continues to grow, our goal is that more patients, and even new SMA patient populations, will be able to experience the transformative impact of this treatment,” Sitra Tauscher-Wisniewski, MD, vice president of clinical development and analytics at Novartis Gene Therapies, said in a statement.

Data from LT-001 demonstrated up to 7.5 years post-dosing durability for children who were treated after presenting symptoms of SMA, who maintained all previously achieved motor milestones. Additionally, during the study, 3 additional individuals achieved the milestone of standing with assistance.

Interim results from the 15-year LT-002 study, which included pre-symptomatic and symptomatic patient populations with intrathecal and IV administration, demonstrated that all individuals maintained motor milestones during their respective parent studies in the follow-up period.

Results from the IV cohort, including 63 individuals, demonstrated how a single dose of onasemnogene abeparvovec provided consistent, durable, and sustained efficacy over time. For those who were pre-symptomatic and in the IV cohort, all children either maintained the highest milestone achieved during the parent study, which was walking alone, or achieved the milestone by data cut-off. Six individuals treated prior to SMA symptom onset and 16 after SMA symptoms achieved new motor milestones in the follow-up period.

All 18 individuals in the LT-002 study who were treated with 1-time investigational OAV101IT, were alive, free from permanent ventilation, and continued to show incremental gains in motor function by the data cut-off in May 2022.

Five of 16 patients who had a milestone assessment achieved new milestones during the long-term follow up, including crawling, standing with assistance, or walking.

Most individuals in LT-002 never received add-on therapy. In the IV cohort, 95% of individuals who were treated before symptom onset achieved the motor milestone of walking alone prior to or without add-on therapy, and 93.8% of individuals treated after SMA symptom onset achieved the milestone of sitting without support prior to or without add-on therapy.

The data will be presented during the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, which will also include, real-world evidence from the RESTORE (NCT04174157) registry.

In the RESTORE registry, investigators reported that adverse events experienced by these individuals were found to be consistent with the previously reported safety findings of onasemnogene abeparvovec, which included acute respiratory failure, dehydration, gastroenteritis, nasopharyngitis, pneumonia, respiratory distress, and viral infection.


Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset. Novartis. News release. March 20, 2023. Accessed March 20, 2023. https://www.novartis.com/news/media-releases/novartis-shares-zolgensma-long-term-data-demonstrating-sustained-durability-75-years-post-dosing-100-achievement-all-assessed-milestones-children-treated-prior-sma-symptom-onset

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