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Ulefnersen - Ionis Pharmaceuticals/Otsuka Pharmaceutical

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Drug Profile

Ulefnersen - Ionis Pharmaceuticals/Otsuka Pharmaceutical

Alternative Names: ION-363; Jacifusen; Ulefnersen sodium

Latest Information Update: 12 Jun 2025

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antisense oligonucleotides; Sclerosants
  • Mechanism of Action Gene silencing; RNA-binding protein FUS expression inhibitors
  • Orphan Drug Status

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

    Yes - Amyotrophic lateral sclerosis
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Amyotrophic lateral sclerosis

Most Recent Events

  • 07 Jun 2025 Efficacy, adverse event and pharmacodynamics data from the expanded access programme in Amyotrophic lateral sclerosis released by Ionis Pharmaceuticals
  • 07 Jun 2025 Ionis Pharmaceuticals initiates a expanded access programme for Amyotrophic lateral sclerosis in USA and Switzerland prior to June 2025
  • 13 Oct 2023 Ulefnersen - Ionis Pharmaceuticals receives Orphan Drug status for Amyotrophic lateral sclerosis in European Union

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