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.

Efficacy and Safety of Vutrisiran in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: Analysis of the East Asian Subpopulation from HELIOS-A

Publication Details

Neurology and Therapy

March 2026

Access Here

Author(s)

Jeeyoung Oh¹, Yoshiki Sekijima², Hideki Mochizuki^{3 4}, Byoung Joon Kim⁵, Ju-Hong Min⁵, Masahisa Katsuno⁶, Cheng Yin Tan⁷, Yi-Chung Lee⁸, Yohei Misumi⁹, Weizhi Zhao¹⁰, Amy Chan-Daniels¹⁰, Chi-Chao Chao¹¹

Affiliations

¹Department of Neurology, Konkuk University Hospital, Seoul, South Korea; ²Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Masumoto, Japan; ³Department of Neurology, Osaka University Graduate School of Medicine, Osaka, Japan; ⁴National Hospital Organization, Osaka Toneyama Medical Center, Osaka, Japan; ⁵Department of Neurology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea; ⁶Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan; ⁷Department of Medicine, University of Malaya, Kuala Lumpur, Malaysia; ⁸Department of Neurology, Taipei Veterans General Hospital, Taipei, Taiwan; ⁹Department of Neurology, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan; ¹⁰Alnylam Pharmaceuticals, Cambridge, MA, USA; ¹¹Department of Neurology, National Taiwan University Hospital, No. 7 Chung Shan S. Rd., Zhongzheng Dist., Taipei City, 100225, Taiwan

Abstract

Introduction:

In the phase 3 HELIOS-A study (NCT03759379), vutrisiran significantly improved a range of disease-related endpoints compared with an external placebo in patients with hereditary transthyretin amyloidosis (ATTRv) with polyneuropathy (ATTRv-PN). This post hoc analysis examined the effects of vutrisiran in East Asian participants of HELIOS-A.

Methods:

HELIOS-A was a global, open-label, phase 3 study in which patients with ATTRv-PN were randomized 3:1 to receive subcutaneous vutrisiran 25 mg every 3 months or intravenous patisiran 0.3 mg/kg every 3 weeks for 18 months. The placebo group of the APOLLO study was used as an external placebo control. The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score + 7 (mNIS + 7) score versus placebo at month 9; secondary endpoints included change from baseline in: Norfolk Quality of Life-Diabetic Neuropathy questionnaire score and 10-meter walk test speed at months 9 and 18, mNIS + 7 score, modified body mass index and Rasch-built Overall Disability Scale score at month 18, and serum transthyretin level through month 18.

Results:

Of the 122 patients receiving vutrisiran in HELIOS-A and 77 patients receiving placebo in APOLLO, 33 were East Asian (vutrisiran: 12, placebo: 21) and had similar baseline characteristics to the overall study cohorts. All outcomes, including the primary endpoint of change from baseline in mNIS + 7 score, showed changes that were consistent in direction and magnitude with the changes seen in the overall HELIOS-A analysis set. Adverse events (AEs) were broadly similar between the East Asian subpopulation and the overall HELIOS-A population; compared with the APOLLO placebo group, fewer patients died or experienced treatment-related AEs and serious AEs.

Conclusions:

Although the number of East Asian patients in HELIOS-A was small, this post hoc analysis showed similar outcomes to those in the overall study populations, indicating that vutrisiran is effective and well tolerated in East Asian patients.