Drug Profile


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

Latest Information Update: 10 Apr 2017

Price : $50

At a glance

  • Originator Isis Pharmaceuticals
  • Developer GlaxoSmithKline; Indiana University School of Medicine; Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action 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

  • 28 Feb 2017 Ionis announces intention to submit NDA to the US FDA for Amyloid polyneuropathy by the end of 2017 if phase III results are positive
  • 09 Aug 2016 Brigham and Women's Hospital, GlaxoSmithKline and Ionis Pharmaceuticals withdraw a phase II trial in Senile systemic amyloidosis and Cardiomyopathy prior to enrolment in USA (NCT02627820)
  • 07 Jul 2016 Interim pharmacodynamics and adverse events data from a phase II trial in Amyloidosis and cardiomyopathy released by Ionis Pharmaceuticals
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