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A study of AVXS 101 in patients with spinal muscular atrophy type 1, 2, 3.

Trial Profile

A study of AVXS 101 in patients with spinal muscular atrophy type 1, 2, 3.

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 05 Aug 2021

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At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Therapeutic Use
  • Acronyms REACH
  • Most Recent Events

    • 03 Aug 2021 According to a Novartis media release, the U.S. Food and Drug Administration (FDA) has lifted partial clinical hold based on data from Novartis comprehensive nonclinical toxicology study in non-human primates (NHP) that addressed all issues identified, including questions of dorsal root ganglia (DRG) injury following IT administration.
    • 03 Aug 2021 Status changed from suspended to recruiting, according to a Novartis media release.
    • 30 Oct 2019 According to a Novartis media release, FDA has placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study. This partial hold by the FDA does not impact marketed Zolgensma or AVXS-101 intravenous (IV) clinical trials. Novartis is working with FDA to determine next steps to release partial hold and resume dosing in the AVXS-101 intrathecal trials.
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