Drug Profile


Alternative Names: ALN-CC5

Latest Information Update: 28 Sep 2017

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Oligonucleotides; Small interfering RNA
  • Mechanism of Action Complement C5 inhibitors; RNA interference
  • 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 Haemolytic uraemic syndrome; Paroxysmal nocturnal haemoglobinuria
  • Preclinical Myasthenia gravis

Most Recent Events

  • 18 Aug 2017 Phase-II clinical trials in Haemolytic uraemic syndrome in Latvia (SC) (EudraCT2017-001082-24)
  • 01 Aug 2017 Alnylam Pharmaceuticals completes a phase I/II trial in Paroxysmal nocturnal haemoglobinuria in United Kingdom and Spain (SC) (NCT02352493)
  • 05 Dec 2016 Sanofi Genzyme elects not to opt-in to develop and commercialise ALN CC5
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