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Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101

Trial Profile

Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 06 Nov 2019

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At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Registrational
  • Acronyms START
  • Sponsors AveXis
  • Most Recent Events

    • 06 Nov 2019 Results assessing the value of onasemnogene abeparvovec gene-replacement therapy in improving survival and motor function and decreasing ventilatory support and hospitalization contrasted to natural history for spinal muscular atrophy type 1 presented at the 22nd Annual European Congress of the International Society for Pharmacoeconomics and Outcomes Research
    • 24 May 2019 According to an AveXis media release, Zolgensma (onasemnogene abeparvovec-xioi) is under regulatory review and anticipated to receive approval in Japan (accelerated Sakigake designation) and the European Union (PRIME designation and reviewed under Accelerated Assessment Procedure) later this year.
    • 24 May 2019 According to an AveXis media release, based on data from the phase 3 STR1VE trial and this phase 1 START trial, the US Food and Drug Administration (FDA) has approved Zolgensma (onasemnogene abeparvovac-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
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