Drug Profile


Alternative Names: ALNTTRsc02

Latest Information Update: 06 Jun 2018

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Heart failure therapies; Small interfering RNA
  • Mechanism of Action Prealbumin inhibitors; RNA interference
  • Orphan Drug Status

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

    Yes - Amyloidosis
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Amyloidosis

Most Recent Events

  • 04 Jun 2018 ALN TTRsc02 receives Orphan Drug status for Amyloidosis in USA
  • 23 Apr 2018 The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issues a positive opinion recommending ALN TTRsc02 for Orphan designation for the treatment of Amyloidosis
  • 28 Mar 2018 Updated efficacy and adverse events data from a phase I trial in Healthy volunteers released by Alnylam Pharmaceuticals
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