Drug Profile


Alternative Names: IONIS-HTTRx; ISIS-HTT Rx

Latest Information Update: 15 Jun 2016

Price : $50

At a glance

  • Originator Isis Pharmaceuticals; Roche
  • Developer Ionis Pharmaceuticals; Roche
  • Class Antisense oligonucleotides
  • Mechanism of Action HD protein inhibitors
  • Orphan Drug Status

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

    Yes - Huntington's disease
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Huntington's disease

Most Recent Events

  • 15 Jun 2016 Phase-II clinical trials in Huntington's disease in United Kingdom, Germany, Canada (Intrathecal) (Ionis Pharmaceuticals pipeline, June 2016)
  • 02 Mar 2016 Preclinical data for transgenic mice and rodent models in Huntington disease released by IONIS Pharmaceuticals
  • 05 Jan 2016 IONIS HTTRx receives Orphan Drug status for Huntington's disease in USA
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