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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: 02 Sep 2026

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Akcea Therapeutics; AstraZeneca; Ionis Pharmaceuticals
  • Class Amides; Amino sugars; Antisense oligonucleotides; Cardiovascular therapies; 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
  • Phase II Cardiomyopathies

Most Recent Events

  • 12 Aug 2026 Ionis Pharmaceuticals in collaboration with AstraZeneca completes a phase III CARDIO-TTRansform trial in Transthyretin-related hereditary amyloidosis in USA, Argentina, Australia, Austria, Belgium, Brazil, Czechia, Canada, Denmark, France, Germany, Greece, Israel, Italy, Japan, Poland, Puerto Rico, Portugal, Spain, Sweden, United Kingdom (SC) (NCT04136171)
  • 09 Jul 2026 Adverse events and efficacy data from a phase III trial in transthyretin-mediated amyloid cardiomyopathy released by AstraZeneca
  • 11 Jun 2026 Phase-II clinical trials in Cardiomyopathies (Combination therapy) in Canada (SC) (NCT07608354)

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