Explore Vutrisiran Data in ATTR‑CM and hATTR‑PN
The efficacy and safety of vutrisiran in ATTR‑CM and hATTR‑PN were established in HELIOS‑B and HELIOS‑A.1,2
HELIOS‑B STUDY DESIGN HELIOS‑B: An international, randomized, double-blind, placebo-controlled, Phase 3 study in 654 patients with ATTR‑CM (wtATTR or hATTR)
aA clinical history of HF was required, with at least 1 previous hospitalization for HF or clinical evidence of HF, with signs and symptoms of volume overload or elevated intracardiac pressures warranting diuretic treatment.1
bRandomization was stratified according to tafamidis use at baseline (yes vs no), ATTR amyloidosis disease type (variant vs wild-type), and NYHA class and age at baseline (NYHA class I or II and age <75 years vs all others).1
cCV hospitalizations or urgent HF visits.1
dThis included up to 6 months of data from the OLE, in which all remaining patients in the placebo arm were eligible to receive vutrisiran.1
e6-MWT and echocardiography measures were not analyzed in the OLE.3
HELIOS‑A STUDY DESIGN HELIOS‑A: An international, randomized, open-label, Phase 3 study in 164 patients with hATTR‑PN
aPrevious use of TTR stabilizers was permitted but patients must have completed a wash-out period (14 days for tafamidis; 3 days for diflunisal) prior to study drug dosing.2
Vutrisiran in hATTR‑PN
Learn more about clinical outcomes with vutrisiran in hATTR‑PN from HELIOS‑A using an interactive tool
Transthyretin Amyloidosis (ATTR)
Learn more about the underlying pathophysiology of ATTR and explore current approaches to diagnosis and managing ATTR‑CM and hATTR‑PN
Alnylam and The Science of RNAi
Explore the science of RNAi and RNAi therapeutics, as well as their features and delivery systems
Publications
Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
New England Journal of Medicine
August 2024
Access NowEfficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial
Amyloid
July 2022
Access NowVutrisiran in Transthyretin Amyloidosis: A Pooled Safety Analysis of HELIOS‑A and HELIOS‑B
JACC: Advances
August 2025
Access Now
References: 1. Fontana M, et al. N Engl J Med. 2025;392:33-44; 2. Adams D, et al. Amyloid. 2023;30:18-26; 3. Garcia-Pavia P, et al. Presented at: European Society of Cardiology Congress; August 29-September 1, 2025; Madrid, Spain.
Abbreviations: 6-MWT, 6-minute walk test; 10-MWT, 10-meter walk test; ACM, all-cause mortality; AE, adverse event; AF, atrial fibrillation; ATTR, transthyretin amyloidosis; ATTR‑CM, transthyretin amyloidosis with cardiomyopathy; CV, cardiovascular; DBP, double-blind period; EQ-5D-5L, EuroQol 5-Dimension 5-Level; hATTR, hereditary transthyretin amyloidosis; hATTR‑PN, hereditary transthyretin amyloidosis with polyneuropathy; HF, heart failure; IV, intravenous; KCCQ-OS, Kansas City Cardiomyopathy Questionnaire-Overall Summary; KPS, Karnofsky Performance Status; mBMI, modified body mass index; mNIS+7, modified Neuropathy Impairment Score +7; NAC, National Amyloidosis Centre; NIS, Neuropathy Impairment Score; Norfolk QOL-DN, Norfolk Quality of Life-Diabetic Neuropathy questionnaire; NT-proBNP, N-terminal prohormone of brain-type natriuretic peptide; NYHA, New York Heart Association; OLE, open-label extension; PND, polyneuropathy disability; q3m, every 3 months; q3w, every 3 weeks; R-ODS, Rasch-built Overall Disability Scale; SC, subcutaneous; TTR, transthyretin.
MED-US-VUTRI-2600004
