Novartis 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.2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry.

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.2 During the time of LT-001, three additional patients also achieved the key milestone of “standing with assistance.”

“I have had the privilege of observing some of the children included in the LTFU studies since they started their Zolgensma clinical trial journey, and the fact that we’re seeing them maintain and, in some cases, gain motor milestones when they are nearly eight years old is truly transformational,” said Dr. Jerry R. Mendell of Nationwide Children’s Hospital. “These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment. I am excited to see the new possibilities that open up to the children, their families and others who may now be able to receive this treatment.”

“Data from the LT-001 and LT-002 studies showed that, regardless of the patient’s symptomatic status at the time of treatment, Zolgensma 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,” said Sitra Tauscher-Wisniewski, MD, Vice President Clinical Development & Analytics, Novartis Gene Therapies.