Novartis: SMA Drug Zolgensma Long-term Data Shows Sustained Durability Up To 7.5 Years Post-dosing

(RTTNews) - Novartis AG (NVS) Monday presented new long-term data of Zolgensma (onasemnogene abeparvovec), the only approved one-time gene therapy for the treatment of spinal muscular atrophy or SMA, demonstrating sustained durability up to 7.5 years post-dosing.

The company noted that latest data from two Long-Term Follow-Up or 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.

Children in LT-001, an ongoing 15-year LTFU study of patients who completed the Phase 1 START study, treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusion.

All children in the presymptomatic intravenous cohort of LT-002 maintained or achieved all assessed motor milestones, including independent walking.

Additionally, children with SMA Type 2 treated with investigational intrathecal OAV101 maintained or achieved new development gains.

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

Novartis noted that more than 3,000 children with spinal muscular atrophy have been treated with Zolgensma to date across clinical trials, managed access programs and in the commercial setting. Zolgensma is now approved in more than 47 countries.

Sitra Tauscher-Wisniewski, MD, Vice President Clinical Development & Analytics, Novartis Gene Therapies, said, "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."

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