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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: 30 May 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

  • Registered Hyperlipoproteinaemia type I
  • Phase III Hypertriglyceridaemia

Most Recent Events

  • 19 May 2025 Ionis Pharmaceuticals announces intention to submit sNDA for Hypertriglyceridemia by 2026
  • 19 May 2025 Efficacy and adverse events data from a phase III trial in Hypertriglyceridaemia released by Ionis Pharmaceuticals
  • 30 Apr 2025 Ionis Pharmaceuticals intends to receive an approve TRYNGOLZA in European Union in second half of 2025

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