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Olezarsen - Ionis Pharmaceuticals

Drug Profile

Olezarsen - Ionis Pharmaceuticals

Alternative Names: AKCEA-APOCIII-LRx; GalNAc3 conjugated antisense oligonucleotide - Ionis Pharmaceuticals; IONIS-APOCIII-LRx; IONISAPOCIII-LRx, sodium salt; ISIS 678354; ISIS-APOCIII-LRx; Olezarsen sodium

Latest Information Update: 16 Apr 2024

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Akcea Therapeutics; Ionis Pharmaceuticals
  • Class Amides; Amino acids; Antihyperlipidaemics; Antisense oligonucleotides; Cardiovascular therapies; Drug conjugates
  • Mechanism of Action Apolipoprotein C III expression inhibitors; Gene silencing
  • Orphan Drug Status

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

    Yes - Hyperlipoproteinaemia type I
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Cardiovascular disorders; Hyperlipoproteinaemia type I; Hypertriglyceridaemia

Most Recent Events

  • 11 Apr 2024 Ionis Pharmaceuticals initiates an expanded-access programme for Hyperlipoproteinaemia type I (NCT06360237)
  • 07 Apr 2024 Efficacy and safety data from the phase III BALANCE trial in Hyperlipoproteinaemia type I released by Ionis Pharmaceuticals
  • 21 Feb 2024 Olezarsen - Ionis Pharmaceuticals receives Breakthrough Therapy status for Hyperlipoproteinaemia type I in USA
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