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ALN CFB

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Drug Profile

ALN CFB

Alternative Names: ALN-CFB

Latest Information Update: 17 Dec 2025

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At a glance

  • Originator Alnylam Pharmaceuticals; Regeneron Pharmaceuticals
  • Developer Regeneron Pharmaceuticals
  • Class Antianaemics; Small interfering RNA
  • Mechanism of Action Complement factor B expression 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 I/II Paroxysmal nocturnal haemoglobinuria

Most Recent Events

  • 06 Nov 2025 Phase-I/II clinical trials in Paroxysmal nocturnal haemoglobinuria in Canada (Parenteral) (NCT07187401)
  • 23 Sep 2025 Preclinical trials in Paroxysmal nocturnal haemoglobinuria in USA (Parenteral) (Prior to September 2025)
  • 23 Sep 2025 Regeneron Pharmaceuticals plans a phase I/II trial for Paroxysmal nocturnal haemoglobinuria and persistent Anemia (Treatment-experienced) (Parenteral) (NCT07187401)

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