Drug Profile


Alternative Names: IONIS-304801; IONIS-APOCIII; IONIS-APOCIIIRx; ISIS-304801; ISIS-APOCIII; ISIS-APOCIII-Rx; Volanesorsen sodium

Latest Information Update: 14 Sep 2017

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At a glance

  • Originator Isis Pharmaceuticals
  • Developer Akcea Therapeutics; Ionis Pharmaceuticals
  • Class Antihyperlipidaemics; Antisense oligonucleotides; Antisense RNA
  • Mechanism of Action Apolipoprotein C-III inhibitors; RNA modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Hyperlipoproteinaemias; Lipodystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Preregistration Hyperlipoproteinaemia type I
  • Phase III Hypertriglyceridaemia; Lipodystrophy
  • Preclinical Cardiovascular disorders

Most Recent Events

  • 12 Sep 2017 Preregistration for Hyperlipoproteinaemia type I in Canada (SC)
  • 12 Sep 2017 Volanesorsen receives priority review status for Hyperlipoproteinaemia type 1 in Canada
  • 31 Aug 2017 Ionis Pharmaceuticals and Akcea Therapeutics plan to make regulatory submissions in Canada in Hyperlipoproteinaemia type I in September 2017
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