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• W3124870890 abstract "<h3>Introduction and Objective</h3> Onasemnogene abeparvovec (formerly AVXS-101), is designed to address the genetic root cause of spinal muscular atrophy (SMA), survival motor neuron 1 gene (<i>SMN1</i>) deletion/mutation. Here, we evaluate final data from STR1VE-US (NCT03306277), a multicenter, open-label, single-arm, single-dose, Phase 3 study conducted in the United States, investigating efficacy and safety of one-time intravenous infusion of onasemnogene abeparvovec in patients with SMA1 (aged, <6 months). <h3>Methods</h3> Co-primary endpoints: independent sitting for ≥30 seconds at the 18 months visit, survival (no death/permanent ventilation) at 14 months of age. Co-secondary endpoints: ability to thrive at 18 months (composite of: tolerates thin liquids, no mechanical nutrition support, maintains weight consistent with age), independence from ventilatory support at 18 months (based on Trilogy BiPAP usage). Safety endpoints: unanticipated treatment-related toxicity of Grade ≥3 based on CTCAE. <h3>Results</h3> All co-primary and co-secondary endpoints demonstrated statistically significant benefits of onasemnogene abeparvovec compared with untreated controls in the Pediatric Neuromuscular Clinical Research (PNCR) study. Thirteen of 22 (59.1%) patients treated with onasemnogene abeparvovec achieved the milestone of functional independent sitting for ≥30 seconds at the 18-month visit (P<0.0001 vs PNCR). Twenty-one of 22 patients (95.5%) survived ≥10.5 months without permanent ventilatory support and 20/22 (90.9%) patients were surviving free of permanent ventilation at 14 months and 18 months of age. For comparison, the relevant PNCR dataset showed event-free survival of 50% at 10.5 months and 25% at 13.6 months. Nine of 22 (40.9%) patients maintained the ability to thrive at 18 months of age, 15/22 (68.2%) patients received no non-oral feeding support at any time during the study, and 18/22 (81.8%) patients were independent of ventilator support at 18 months of age based on Trilogy BiPAP data. Patients exhibited rapid (1-month post doing) and sustained improvements in motor function (CHOP INTEND scores) and achieved motor milestones, previously unseen in PNCR dataset. Adverse events were manageable and consistent with the known safety profile of onasemnogene abeparvovec. <h3>Conclusions</h3> In the STR1VE-US study, patients with SMA1 treated with onasemnogene abeparvovec demonstrated a significant therapeutic benefit and a favourable the benefit-risk profile." @default.
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• W3124870890 date "2021-01-21" @default.
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• W3124870890 title "S12 Onasemnogene abeparvovec gene therapy for spinal muscular atrophy type 1: phase 3 study (STR1VE-US)" @default.
• W3124870890 doi "https://doi.org/10.1136/thorax-2020-btsabstracts.18" @default.
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