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Drug Profile


Alternative Names: AKCEA-TTR-LRx; ION-682884; ION-TTR-LRx; IONIS-TTR-LRx

Latest Information Update: 14 Jan 2020

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Akcea Therapeutics; Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action Prealbumin expression inhibitors
  • 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 Transthyretin-related hereditary amyloidosis

Most Recent Events

  • 10 Jan 2020 Ionis Pharmaceuticals in collaboration with Akcea Therapeutics initiates enrolment in the phase III CARDIO-TTRansform trial for Transthyretin related hereditary amyloidosis in USA (SC) (NCT04136171)
  • 24 Nov 2019 Phase-III clinical trials in Transthyretin-related hereditary amyloidosis in USA (SC) (NCT04136184)
  • 06 Nov 2019 Ionis Pharmaceuticals plans the phase III CARDIO-TTRansform trial in Transthyretin‑mediated amyloid cardiomyopathy in fourth quarter of 2019 (ION-682884-CS2; EudraCT2019-002835-27; NCT04136171)
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