CONTEXT: Novrtis going old school with the announcement of a global Phase 3b study in children with spinal muscular atrophy (SMA). Respect to Novartis for making the announcement and calling it a Phase 3b – this author hasn’t heard that term in a very long time. Enrollment in the study is expected to start in September of 2021 and will run in North America, Europe, Australia and Taiwan.
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1. “About SMART. SMART is a Phase 3b, open-label, single-arm, multicenter study designed to evaluate the safety, tolerability and efficacy of a one-time IV infusion of Zolgensma in pediatric patients who have symptomatic SMA with bi-allelic mutations in the SMN1 gene and any copy number of the SMN2 gene and weigh ≥ 8.5 kg and ≤ 21 kg.”
2. “Basel, April 23, 2021 – Novartis today announced plans to initiate SMART, a Phase 3b clinical study to evaluate the safety and efficacy of Zolgensma® (onasemnogene abeparvovec) in young children with spinal muscular atrophy (SMA) weighing ≥ 8.5 kg and ≤ 21 kg, following a one-time, intravenous (IV) infusion.”
3. “The Zolgensma clinical program has studied symptomatic children less than six months of age with SMA Type 1 in the clinical setting, but emerging findings from the RESTORE registry, recently presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference, indicate older children (≥ 6 months) achieved a clinically meaningful benefit when treated with Zolgensma alone, after switching to gene therapy or in combination with another SMA therapy, with safety events consistent with the previously described safety profile.”
4. “To date, more than 1,000 patients have been treated with Zolgensma across clinical trials, managed access programs, and in the commercial setting.1. Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children’s Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self- complementary DNA technology for the treatment of SMA.”
5. “About Spinal Muscular Atrophy (SMA) . SMA is the leading genetic cause of infant death.2,3 If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.4 SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.2 It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.5,6 This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly.”