Drug Profile



Latest Information Update: 09 Jun 2017

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Akcea Therapeutics; Ionis Pharmaceuticals
  • Class Antihyperlipidaemics; Antisense oligonucleotides
  • Mechanism of Action ANGPTL3 protein inhibitors
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Hyperlipoproteinaemia type IIa

Most Recent Events

  • 08 Aug 2017 Akcea plans a phase II trial for Non-alcoholic fatty liver disease and Hyperlipidemias in the second half of 2017
  • 25 May 2017 Updated efficacy and adverse events data from a phase I/II trial in Liver disorders released by Akcea Therapeutics
  • 01 Mar 2017 Ionis Pharmaceuticals plans a clinical trial for Liver disorders (non-alcoholic fatty liver disease or non-alcoholic steatohepatitis) (Ionis Pharmaceuticals 10-K, March 2017)
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