Vutrisiran - Alnylam Pharmaceuticals
Alternative Names: ALN-65492; ALN-TTRsc02; AMVUTTRALatest Information Update: 16 Jun 2025
At a glance
- Originator Alnylam Pharmaceuticals
- Class Amides; Amino sugars; Drug conjugates; Pyrrolidines; Small interfering RNA
- Mechanism of Action Prealbumin expression inhibitors; RNA interference
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Orphan Drug Status
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
- 09 Jun 2025 European Commission approves Vutrisiran (AMVUTTRA®) for treatment of Transthyretin-related hereditary amyloidosis with cardiomyopathy (ATTR-CM) in European Union, Iceland, Liechtenstein, and Norway
- 31 May 2025 European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) adopts a positive opinion on the maintenance of the EU Orphan Designation for Vutrisiran (AMVUTTRA®) in ATTR amyloidosis
- 17 May 2025 Alnylam Pharmaceuticals expects a formal regulatory decision by the European Commission of the EMA by the third quarter of 2025 for the use of vutrisiran for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults