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Eplontersen - AstraZeneca/Ionis Pharmaceuticals

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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; WAINZUA

Latest Information Update: 18 Mar 2025

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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

  • 10 Mar 2025 Registered for Transthyretin-related hereditary amyloidosis in European Union, Norway, Iceland, Liechtenstein (SC)
  • 15 Nov 2024 AstraZeneca completes a phase I pharmacokinetics, pharmacodynamics and safety trial in healthy chinese volunteers in China (SC, Injection) (NCT06527755)
  • 07 Nov 2024 Registered for Transthyretin-related hereditary amyloidosis in United Kingdom (SC)

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