Drug Profile


Alternative Names: GSK 2998728; IONIS TTRRx; ISIS 420915; ISIS-GSK1Rx; ISIS-TTRRx

Latest Information Update: 23 Aug 2017

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At a glance

  • Originator Isis Pharmaceuticals
  • Developer GlaxoSmithKline; Indiana University School of Medicine; Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action Antisense RNA modulators; Protein synthesis inhibitors
  • Orphan Drug Status

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

    Yes - Amyloid polyneuropathy
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Amyloid polyneuropathy
  • Phase II Amyloidosis; Cardiomyopathies

Most Recent Events

  • 11 Aug 2017 Ionis completes a phase III trial for Amyloid polyneuropathy in USA, Argentina, Brazil, France, Germany, Italy, New Zealand, Portugal, Spain and the United Kingdom (NCT01737398)
  • 11 Aug 2017 Ionis Pharmaceuticals announces intention to submit MAA to the EMA for Amyloid polyneuropathy by the end of 2017
  • 11 Aug 2017 GlaxoSmithKline declines options to licence inotersen
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