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Eplontersen - AstraZeneca/Ionis Pharmaceuticals

Drug Profile

Eplontersen - AstraZeneca/Ionis Pharmaceuticals

Alternative Names: AKCEA-TTR-LRx; Eplontersen sodium - AstraZeneca/Ionis Pharmaceuticals; ION-682884; ION-TTR-LRx; IONIS-TTR-LRx; WAINUA

Latest Information Update: 29 Feb 2024

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Akcea Therapeutics; AstraZeneca; Ionis Pharmaceuticals
  • Class Amides; Amino sugars; Antisense oligonucleotides; Drug conjugates
  • Mechanism of Action Gene silencing; Prealbumin expression inhibitors
  • Orphan Drug Status

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

    Yes - Transthyretin-related hereditary amyloidosis
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Transthyretin-related hereditary amyloidosis

Most Recent Events

  • 08 Feb 2024 Eplontersen - AstraZeneca/Ionis Pharmaceuticals receives Fast Track designation for Transthyretin-related hereditary amyloidosis [SC] (In adults) in USA
  • 08 Feb 2024 Ionis and AstraZeneca seek regulatory approval for eplontersen in Europe and other parts of the world for transthyretin-related hereditary amyloidosis
  • 22 Jan 2024 Launched for Transthyretin-related hereditary amyloidosis (In adults) in USA (SC)
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