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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: 11 Dec 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 Dry macular degeneration; Myasthenia gravis; Paroxysmal nocturnal haemoglobinuria
  • Phase II Haemolytic uraemic syndrome; IgA nephropathy

Most Recent Events

  • 08 Dec 2024 Efficacy and adverse events data from a phase III ACCESS-1 trial in Paroxysmal nocturnal hemoglobinuria released by Regeneron Pharmaceuticals
  • 08 Dec 2024 Efficacy and adverse events data from a phase III OLE(ACCESS-EXTENSION) trial in Paroxysmal nocturnal hemoglobinuria released by Regeneron Pharmaceuticals
  • 30 Oct 2024 Phase-III clinical trials in Dry macular degeneration (Combination therapy) in USA (SC) (NCT06541704)

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