Vutrisiran: AL Amyloidosis or Leptomeningeal Amyloidosis

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The full Prescribing Information for AMVUTTRA® (vutrisiran) is provided here. Alnylam Pharmaceuticals does not recommend the use of its products in any manner that is inconsistent with the approved Prescribing Information. This resource may contain information that is not in the approved Prescribing Information.

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Summary

 The use of vutrisiran in patients with primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis has not been evaluated in clinical studies.

In the phase 3 HELIOS-A and HELIOS-B studies, patients were excluded if they had known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis.1–4

AMVUTTRA Prescribing Information – Relevant Content

The INDICATIONS AND USAGE section provides the following information5:

Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis

AMVUTTRA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

Cardiomyopathy of Wild-type or Hereditary Transthyretin-mediated Amyloidosis

AMVUTTRA is indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.

Abbreviations

AL = amyloid light chain; ATTR-CM = transthyretin amyloidosis with cardiomyopathy; hATTR-PN = hereditary transthyretin amyloidosis with polyneuropathy.

Updated 8 October 2025

References

1. Adams D, Tournev IL, Taylor MS, et al. Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid. 2023;30(1):18-26. doi:10.1080/13506129.2022.2091985

2. Alnylam Pharmaceuticals. Data on file. MED-ALL-TTRSC02-2300015.

3. Fontana M, Berk JL, Gillmore JD, et al. Vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy. N Engl J Med. 2025;392(1):33-44. doi:10.1056/NEJMoa2409134

4. Protocol for: Fontana M, Berk JL, Gillmore JD, et al. Vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy. N Engl J Med. 2025;392(1):33-44. doi:10.1056/NEJMoa2409134

5. AMVUTTRA (vutrisiran) Prescribing Information. Cambridge, MA: Alnylam Pharmaceuticals, Inc.


MED-ALL-VUTRI-2500050 1.0 Approved through Oct 2027

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