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Vutrisiran - Alnylam Pharmaceuticals

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Drug Profile

Vutrisiran - Alnylam Pharmaceuticals

Alternative Names: ALN-65492; ALN-TTRsc02; AMVUTTRA

Latest Information Update: 25 Feb 2026

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Amides; Amino sugars; Drug conjugates; Small interfering RNA
  • Mechanism of Action Prealbumin expression inhibitors; RNA interference
  • Orphan Drug Status

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

    Yes - Transthyretin-related hereditary amyloidosis
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Transthyretin-related hereditary amyloidosis

Most Recent Events

  • 16 Feb 2026 Preregistration for Transthyretin-related hereditary amyloidosis in South Korea, Taiwan, Hong Kong, Serbia, Macedonia, Kuwait (SC)
  • 16 Dec 2025 Health Canada approves Vutrisiran for treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in Canada
  • 08 Nov 2025 Efficacy data from a phase III HELIOS-B trial in Transthyretin amyloidosis presented at the American Heart Association Scientific Sessions 2025 (AHA-2025)

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