SMA – THERAPY: P.258 Onasemnogene aveparvovec gene therapy for spinal muscular atrophy type 1 (SMA1): Phase 3 study update (STR1VE-EU)

Abstract

Onasemnogene abeparvovec is a one-time intravenous gene therapy designed to address the genetic root cause of SMA1, survival motor neuron 1 gene (SMN1) deletion. We report preliminary data from the ongoing phase 3 STR1VE-EU (2017-000266-29/NCT03461289) study, evaluating efficacy/safety of onasemnogene abeparvovec infusion. STR1VE-EU is a multicenter, open-label, single-arm, single-dose study in symptomatic patients (pts) with SMA1 aged <6 months (mos; biallelic SMN1 mutations, 1–2xSMN2). Outcomes: independent sitting ≥10 seconds (sec) throughout 18 mos; survival (no death/permanent ventilation) at 14 mos.