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Pelacarsen - Ionis Pharmaceuticals/Novartis

Drug Profile

Pelacarsen - Ionis Pharmaceuticals/Novartis

Alternative Names: AKCEA-APO(a)-LRx; IONIS-681257; IONIS-APO(a)-LRx; IONIS-APO-LRx; ISIS 681257; ISIS-APO(a)-LRx; ISIS-APOALRx; Pelacarsen sodium; TQJ 230

Latest Information Update: 28 Mar 2024

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At a glance

  • Originator Isis Pharmaceuticals
  • Developer Novartis; The Chinese University of Hong Kong
  • Class Antihyperlipidaemics; Antisense oligonucleotides
  • Mechanism of Action Apolipoprotein A inhibitors; Apolipoproteins A expression inhibitors; Gene silencing; RNA interference
  • 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 III Cardiovascular disorders; Hyperlipoproteinaemia
  • Phase II Aortic valve stenosis

Most Recent Events

  • 07 Mar 2024 Phase-II clinical trials in Aortic valve stenosis in United Kingdom, Israel (SC) (NCT05646381)
  • 20 Feb 2024 Novartis Pharmaceuticals plans a phase III trial in Hyperlipoproteinaemia in USA (SC) in May 2024 (NCT06267560)
  • 29 Sep 2023 Novartis Pharmaceuticals initiates a phase III trial in Hyperlipoproteinemia in Germany (SC) (NCT05900141)
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