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Cemdisiran - Alnylam Pharmaceuticals

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Drug Profile

Cemdisiran - Alnylam Pharmaceuticals

Alternative Names: AD-62643; ALN 62643; ALN-CC5

Latest Information Update: 23 Aug 2024

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Developer Alnylam Pharmaceuticals; Regeneron Pharmaceuticals
  • Class Drug conjugates; 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.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Myasthenia gravis; Paroxysmal nocturnal haemoglobinuria
  • Phase II Haemolytic uraemic syndrome; IgA nephropathy

Most Recent Events

  • 07 Aug 2024 Regeneron Pharmaceuticals plans a phase III SIENNA trial for Geographic atrophy (Monotherapy, Combination therapy) in October 2024 (NCT06541704) (SC, Injection)
  • 28 Jun 2024 Austin Neuromuscular Center plans an early phase I trial for Inclusion body myositis (Combination therapy) in USA (SC, Injection) (NCT06479863)
  • 13 Jun 2024 Updated efficacy and adverse events data from a phase II trial in Paroxysmal nocturnal haemoglobinuria presented at the 29th Congress of the European Haematology Association (EHA-2024)

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