Vutrisiran - Alnylam Pharmaceuticals
Alternative Names: ALN-65492; ALN-TTRsc02; AMVUTTRALatest Information Update: 03 Sep 2025
At a glance
- Originator Alnylam Pharmaceuticals
- Class Amides; Amino sugars; Drug conjugates; Small interfering RNA
- Mechanism of Action Prealbumin expression inhibitors; RNA interference
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Orphan Drug Status
Yes - Transthyretin-related hereditary amyloidosis
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- 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
- 29 Aug 2025 Efficacy data from the phase-III HELIOS-B trial in Transthyretin-related hereditary amyloidosis presented at the Annual Congress of the European Society of Cardiology together with World Congress of Cardiology (ESC-Card-WCC-2025)
- 11 Aug 2025 Registered for Transthyretin-related hereditary amyloidosis in United Arab Emirates (SC)