Drug Profile


Alternative Names: ALN-CC5

Latest Information Update: 20 May 2017

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Oligonucleotides; Small interfering RNA
  • Mechanism of Action Complement C5 inhibitors
  • Orphan Drug Status

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

  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Phase II Paroxysmal nocturnal haemoglobinuria
  • Preclinical Haemolytic uraemic syndrome; Myasthenia gravis

Most Recent Events

  • 05 Dec 2016 Sanofi Genzyme elects not to opt-in to develop and commercialise ALN CC5
  • 05 Dec 2016 Updated pharmacodynamics, adverse events and drug interactions data from a phase I/II trial in Paroxysmal nocturnal haemoglobinuria presented at the 58th Annual Meeting and Exposition of the American Society of Hematology (ASH-Hem-2016)
  • 23 Nov 2016 Alnylam terminates a phase II trial in Paroxysmal nocturnal haemoglobinuria (Treatment experienced) in Spain (SC) (EudraCT2016-002943-40)
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