Drug Profile


Alternative Names: ALN-TTRsc; SAR438714; siTTRsc

Latest Information Update: 24 Mar 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

  • Discontinued Amyloid polyneuropathy; Cardiomyopathies

Most Recent Events

  • 08 Feb 2017 Alnylam Pharmaceuticals terminates a phase III extension trial for familial amyloidotic Cardiomyopathy (In the elderly, In adults) due to safety findings in United Kingdom (SC) (EudraCT2015-005333-49)
  • 01 Feb 2017 Alnylam Pharmaceuticals completes a phase II trial in Amyloid polyneuropathy in France, Germany, Portugal, Sweden, United Kingdom (SC) (NCT02595983)
  • 01 Nov 2016 Alnylamt terminates a phase III trial in Cardiomyopathy (In adults, In the elderly) due to unacceptable risk benefit profile in USA, United Kingdom, Belgium, Canada, France, Germany, Italy, Sweden and Spain (SC) (NCT02319005)
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