Publications

The material below is provided to support scientific exchange. Any content about investigational therapeutics or investigational uses of locally approved products does not establish the safety or efficacy of these therapeutics or uses, and there is no guarantee of local regulatory approval.

For questions beyond a product’s authorized indications or for information not available here, please contact Alnylam Medical Information.

Vutrisiran in East Asian Patients With Transthyretin Amyloidosis With Cardiomyopathy: Post-Hoc Analysis of HELIOS-B

Publication Details

JACC: Asia

May 2026

Access Here

Author(s)

Jin Endo¹, Tetsuo Minamino², Jaewon Oh³, Nobuhiro Tahara⁴, Kenichi Tsujita⁵, Chisato Izumi⁶, Takahiro Okumura⁷, Kenji Onoue⁸, Hiroaki Kitaoka⁹, Chongshu Chen¹⁰, Amy Chan-Daniels¹⁰, Darae Kim¹¹

Affiliations

¹Department of Cardiology, Keio University School of Medicine, Tokyo, Japan; ²Department of Cardiorenal and Cerebrovascular Medicine, Kagawa University, Kita-gun, Japan; ³Division of Cardiology, Severance Hospital, Yonsei University Health System, Seoul, South Korea; ⁴Division of Cardiovascular Medicine, Department of Medicine, Kurume University School of Medicine, Kurume, Japan; ⁵Department of Cardiovascular Medicine, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan; ⁶Department of Heart Failure and Transplantation, National Cerebral and Cardiovascular Center, Suita, Japan; ⁷Department of Cardiology, Nagoya University Graduate School of Medicine, Nagoya, Japan; Department of Advanced Cardiovascular Therapeutics, Nagoya University Graduate School of Medicine, Nagoya, Japan; ⁸Department of Cardiovascular Medicine, Nara Medical University, Kashihara, Japan; ⁹Department of Cardiology and Geriatrics, Kochi University, Kochi, Japan; ¹⁰Clinical Science, Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA; ¹¹Division of Cardiology, Department of Medicine, Heart Vascular Stroke Institute, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

Abstract

Background:

In the phase III Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (HELIOS-B), vutrisiran significantly reduced all-cause mortality and recurrent cardiovascular events and preserved functional capacity and quality of life. However, data in East Asian populations remain limited. The authors aimed to assess the efficacy and safety of vutrisiran in East Asian patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) enrolled in HELIOS-B.

Methods:

This post-hoc analysis included 32 patients from Japan and South Korea randomized to vutrisiran (n = 17) or placebo (n = 15). The primary endpoint was a composite of all-cause mortality and recurrent cardiovascular events for up to 36 months. Secondary endpoints were changes from baseline to month 30 in the 6-minute walk test distance, Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score, and New York Heart Association (NYHA) functional class. Exploratory endpoints included changes in N-terminal pro–B-type natriuretic peptide and troponin I. Safety was also evaluated.

Results:

Baseline characteristics were generally similar to the overall population, although baseline tafamidis use was lower in East Asian patients. Vutrisiran reduced the risk of the composite primary endpoint vs placebo (HR: 0.20; 95% CI: 0.04-0.93). Vutrisiran attenuated declines in 6-minute walk test distance and KCCQ-OS scores, and a greater proportion of patients maintained or improved NYHA functional class. Smaller increases in N-terminal pro–B-type natriuretic peptide and troponin I levels were observed with vutrisiran vs placebo. Similar trends were observed in patients not receiving baseline tafamidis. Safety was consistent with the overall population.