Pivotal trial of AVXS 101 in patients with spinal muscular atrophy type 1

Trial Profile

Pivotal trial of AVXS 101 in patients with spinal muscular atrophy type 1

Planning
Phase of Trial: Phase II

Latest Information Update: 08 Feb 2017

At a glance

  • Drugs AVXS 101 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Therapeutic Use
  • Most Recent Events

    • 08 Feb 2017 New trial record
    • 06 Feb 2017 According to an Avexis media release, this trial is expected to initiate in the second half of 2017.
    • 06 Feb 2017 According to Avexis media release, this trial will reflect single-arm design and use natural history of the disease as a comparator updated based on the receipt of the Scientific Advice response from the Scientific Advice Working Party within the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
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