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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: 03 Sep 2025

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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

  • 31 Aug 2025 Adverse events and efficacy data from the phase-III HELIOS-B trial and it's open-label extension in Transthyretin-related hereditary amyloidosis released by Alnylam Pharmaceuticals
  • 11 Aug 2025 Registered for Transthyretin-related hereditary amyloidosis in United Arab Emirates (SC)
  • 31 Jul 2025 Japanese Pharmaceuticals and Medical Devices Agency (PMDA) approves Vutrisiran (AMVUTTRA®) for treatment of Transthyretin-related hereditary amyloidosis with cardiomyopathy in Japan (ATTR-CM)

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