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Olezarsen - Ionis Pharmaceuticals

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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; TRYNGOLZA

Latest Information Update: 04 Dec 2025

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Amides; Amino acids; Antihyperlipidaemics; Antisense oligonucleotides; 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

  • Marketed Hyperlipoproteinaemia type I
  • Phase III Hypertriglyceridaemia

Most Recent Events

  • 01 Dec 2025 Olezarsen - Ionis Pharmaceuticals receives Breakthrough Therapy status for Hypertriglyceridaemia in USA
  • 08 Nov 2025 Sobi plans to submit a marketing authorisation application for Hyperlipoproteinemia Type I to EMA in 2026
  • 08 Nov 2025 Ionis Pharmaceuticals plans to launch olezarsen, in 2026

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