Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update

Research Question: SMA1 is a rapidly progressing neurologic disease caused by biallelic survival motor neuron 1 gene (SMN1) deletion/mutation. The one-time SMN GRT onasemnogene abeparvovec (AVXS-101) addresses the genetic root cause of SMA. In a phase 1/2a study (NCT02122952), AVXS-101 resulted in exceptional event-free survival, motor function improvements, and motor milestone achievement in patients with SMA1. We report data from the multicenter, open-label, pivotal phase 3 STR1VE study (NCT03306277) investigating intravenous AVXS-101 in SMA1 patients (bi-allelic SMN1 mutations/deletions, 2xSMN2) aged