Amvuttra (vutrisiran) / Alnylam, Sanofi, Royalty - LARVOL DELTA

Evaluation of Vutrisiran in ATTR Amyloidosis with Cardiomyopathy (ASHP 2025) - No abstract available

Amyloidosis • Cardiomyopathy • Cardiovascular

From Stabilization to Silencing: Assessing Adherence and Outcomes of Tafamidis and Vutrisiran in Patients with Cardiac Amyloidosis in a Rural Setting (ASHP 2025) - No abstract available

Adherence • Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiovascular

Stabilizers, Silencers, and Disease Stage: Lessons From Recent Trials of ATTR Cardiac Amyloidosis. (PubMed, J Am Coll Cardiol) - No abstract available

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

REPLY: Vutrisiran in ATTR-CM: Questions on Combination Therapy and Early Biomarker Kinetics. (PubMed, J Am Coll Cardiol) - No abstract available

Biomarker • Journal

Vutrisiran in ATTR-CM: Questions on Combination Therapy and Early Biomarker Kinetics. (PubMed, J Am Coll Cardiol) - No abstract available

Biomarker • Journal

Class-Specific Adverse Events of Patients Treated with Small Interfering RNA Therapeutics: A Disproportionality Analysis of the United States Food and Drug Administration Adverse Event Reporting System Database Based on the MY FAERS Platform. (PubMed, Nucleic Acid Ther) - "This study aimed to identify and quantify CAE-siRNA associated with U.S. Food and Drug Administration (FDA)-approved siRNA drugs (patisiran, givosiran, vutrisiran, inclisiran, and lumasiran) using real-world pharmacovigilance data, focusing on potential class-wide effects. This study identified clinically relevant CAE-siRNA, particularly hepatic toxicity for inclisiran, supporting enhanced monitoring. While disproportionality analyses are hypothesis generating, these findings underscore the need for targeted pharmacovigilance to optimize the safety of this promising drug class."

Adverse events • Journal • Back Pain • Fatigue • Gastroenterology • Gastrointestinal Disorder • Musculoskeletal Pain • Pain

Cost-effectiveness of vutrisiran, tafamidis, and their combination in amyloid transthyretin cardiomyopathy. (PubMed, Eur Heart J Qual Care Clin Outcomes) - No abstract available

HEOR • Journal • Cardiomyopathy • Cardiovascular

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) (clinicaltrials.gov) - P3 | N=164 | Completed | Sponsor: Alnylam Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Amyloidosis • Cardiac Amyloidosis

NICE recommends vutrisiran for adults with ATTR-CM in final draft guidance (PharmaTimes) - "Around 1,500 patients across England and Wales could be eligible for the treatment, which is the first and only long-acting disease-modifying therapy to treat both ATTR-CM and hereditary ATTR with stage 1 or 2 polyneuropathy."

NICE • Amyloidosis • Cardiomyopathy

Relative efficacy of tafamidis, acoramidis, patisiran and vutrisiran in patients with transthyretin cardiac amyloidosis: a network meta-analysis. (PubMed, Eur Heart J Open) - "When the ATTR-ACT trial was excluded from the analysis, vutrisiran monotherapy consistently showed the highest probability of being ranked better than other treatments in terms of primary end-point. Although differences in trial design and study populations complicate direct efficacy comparisons, tafamidis demonstrated the highest efficacy in improving survival, reducing cardiovascular hospitalizations, and enhancing functional capacity and quality of life in patients with ATTR-CA, but also vutrisiran and acoramidis emerged as viable options."

Journal • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Real-world effectiveness of targeted therapies in ATTR cardiomyopathy: A meta-analysis integrating population-based data. (PubMed, ESC Heart Fail) - "Targeted treatments for ATTR-CM significantly reduce mortality and cardiovascular hospitalizations. When extrapolated to population-level data, these treatments show clinical benefits, emphasizing the importance of early diagnosis and therapeutic intervention."

Journal • Real-world evidence • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Timing of Mortality Benefit in Outcomes Trials in Transthyretin Amyloidosis. (PubMed, J Am Coll Cardiol) - "TTR silencers and stabilizers in ATTR-CM confer a delayed but consistent mortality benefit, with no significant differences observed between the three major trials. This uniform pattern may reflect a shared mechanism of action-reducing new amyloid deposition rather than reversing established disease, and underscore the importance of early treatment initiation and adequate trial duration to capture delayed mortality effects."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

LS.01: Introduction to AMVUTTRA® (vutrisiran) (AHA 2025) - "Description: Join our distinguished faculty as they review the clinical profile of AMVUTTRA for the treatment of the cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM) in adults. Sponsored By: Alnylam Pharmaceuticals, Inc.This event is not part of the official Scientific Sessions Conference 2025 as planned by the American Heart Association Committee on Scientific Sessions Programming"

Amyloidosis • Cardiomyopathy • Cardiovascular

Vutrisiran Improved Outcomes Versus Placebo in Patients with Transthyretin Amyloidosis with Cardiomyopathy and Severe Chronic Kidney Disease: Post Hoc Analysis of HELIOS-B (AHA 2025) - P3 | "Outcomes were assessed overall and by baseline tafamidis use (monotherapy and baseline tafamidis subgroups). Median (IQR) eGFR at baseline in pts receiving vutrisiran and PBO was 64 (50–81) and 65 (53–81) mL/min/1.73m2, respectively. Vutrisiran appeared to preserve renal function in pts with ATTR-CM. Consistent with results from the overall population, vutrisiran reduced the risk of ACM and CV events vs PBO in pts with ATTR-CM and advanced CKD in HELIOS-B; results require corroboration in a larger pt population."

Clinical • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Chronic Kidney Disease • Nephrology • Renal Disease

Patterns of Treatment Adherence and Administration Preferences for People With Transthyretin Amyloid Cardiomyopathy: A Real-World Survey in Europe, the United States, and Japan (ISPOR-EU 2025) - "At survey, 73% of patients were prescribed treatment, of which 93% were prescribed DMTs including tafamidis (85%), patisiran (6%), vutrisiran (2%) and diflunisal (2%). Tafamidis was the most frequently prescribed DMT, and adherence was high. Patients more frequently reported feeling comfortable with oral medications than injections/infusions, particularly once daily orals, underscoring the relevance of administration preferences among treatment landscapes. Further research is needed to explore outcomes with ATTR treatments."

Adherence • Clinical • Real-world • Real-world evidence • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Comparative Efficacy and Safety of Vutrisiran, Acoramidis, Tafamidis in Transthyretin Amyloid Cardiomyopathy: A Network Meta-Analysis (AHA 2025) - "Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, life-threatening disorder caused by transthyretin deposition in cardiac tissue. Vutrisiran demonstrated the most consistent survival and biomarker benefits, while Patisiran ranked highest in overall efficacy. Acoramidis showed strong improvement in functional capacity but varied across domains."

Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Transthyretin Cardiac Amyloidosis Evaluation and Management: 2025 ACC Concise Clinical Guidance. (PubMed, J Am Coll Cardiol) - "Finally, individuals with transthyretin amyloid cardiomyopathy have benefitted from advances in broadly effective heart failure therapies, namely mineralocorticoid receptor antagonists and sodium glucose-cotransporter 2 inhibitors, as well as specific disease-modifying therapies with transthyretin stabilizers, tafamidis and acoramidis, and the transthyretin silencer vutrisiran. The purpose of this Concise Clinical Guidance is to offer updated strategies to clinicians, reflecting the expanding therapeutic landscape, and reinforcing best practices for the diagnosis and management of transthyretin amyloid cardiomyopathy with a focus on choice of disease-modifying therapies, heart failure therapies, and future directions."

Clinical guideline • Journal • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Heart Failure • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Transplantation

Coramitug, a Humanized Monoclonal Antibody for the Treatment of Transthyretin Amyloid Cardiomyopathy: a Phase 2, Randomized, Multicenter, Double-Blind, Placebo-Controlled Trial. (PubMed, Circulation) - "This phase 2 trial showed that coramitug, an antibody targeting misfolded transthyretin in ATTR-CM, was well tolerated and at a dose of 60 mg/kg resulted in a statistically significant reduction in NT-proBNP, a validated marker of disease progression, with no statistically significant effect on 6MWT within 52 weeks."

Journal • P2 data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

RNA Interference Therapeutics for Hereditary Transthyretin-Mediated Amyloidosis with Neuropathy: A Systematic Review (ASH 2024) - "Currently, limited treatment options are available for ATTRv, which include orthotopic liver transplantation and transthyretin tetramer stabilizers (tafamidis or diflunisal)...The most common cause of death reported was cardiac failure.Conclusion : RNA interference therapies, including Eplontersen, Inotersen, Vutrisiran, and Patisiran, have demonstrated significant benefits in enhancing the quality of life for patients with ATTRv and associated polyneuropathy...While RNAi therapies offer substantial symptomatic relief, vigilant surveillance for side effects is crucial to optimize patient outcomes and ensure safety. Further studies, including comparative studies, are essential to establish therapeutic protocols and ensure improved quality of life for patients with ATTRv."

Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Diabetic Neuropathy • Glomerulonephritis • Heart Failure • Hematological Disorders • Lupus Nephritis • Nephrology • Pain • Renal Disease • Thrombocytopenia

Transthyretin Amyloid Cardiomyopathy: The Plot Thickens as Novel Therapies Emerge. (PubMed, US Cardiol) - "Finally, we explore key unresolved questions, including the need for head-to-head comparative trials, the potential role of combination therapy, and the optimal timing for initiating treatment as earlier recognition becomes more common. As novel therapies gain approval this review serves as a timely, focused resource to support clinicians managing ATTR-CM."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Neurological efficacy and safety of RNA therapeutics in hereditary transthyretin amyloidosis: a systematic review and meta-analysis of randomized controlled trials. (PubMed, Ther Adv Cardiovasc Dis) - "RNA therapeutics are effective and well-tolerated in patients with hATTR, significantly improving quality of life and the progression of neurological impairment. siRNAs demonstrate better outcomes compared to ASOs."

Journal • Retrospective data • Review • Amyloidosis • Cardiac Amyloidosis • Diabetic Neuropathy • Gastrointestinal Disorder • Pain

New Post Hoc Analysis from the HELIOS-B Phase 3 Study Shows Vutrisiran Improved Measures of Heart Structure and Function in Patients with ATTR-CM (Businesswire) - "Data...were presented at the American Heart Association (AHA) Scientific Sessions 2025....Treatment with vutrisiran also reduced extracellular volume (ECV), which is thought to reflect amyloid buildup in the heart. At Year 3, amyloid regression, as assessed by ECV, was observed in 22% of patients treated with vutrisiran, while no patients who received placebo showed regression; conversely, progression occurred in 63% of patients who received placebo compared to 11% of patients treated with vutrisiran. At Year 3, patients treated with vutrisiran exhibited an absolute mean (standard deviation) reduction in ECV of -0.10% (± 4.72) versus an increase of 7.86% (± 5.67) in the placebo group (p=0.006)."

P3 data • Cardiac Amyloidosis • Cardiomyopathy

Royalty Pharma acquires AMVUTTRA royalty for $310 million (Streetinsider.com) - "The transaction covers AMVUTTRA sales beginning October 1, 2025, excluding fixed payments from the original Blackstone investment of $70 million....projects sales to exceed $6 billion by 2028."

Commercial • Sales projection • Amyloidosis • Cardiomyopathy

Vutrisiran Healthcare Resource Utilization, Costs, Discontinuation, and Mortality: a Retrospective Database Analysis (AHA 2025) - "One-third to half of pts presented to the hospital within 1.5 y of vutrisiran treatment (before ATTR-CM approval); within 12 mo, roughly 1 in 4 died or discontinued therapy for ≥90 d past the 90-d administration schedule, with many switching to other ATTR treatments. This suggests that vutrisiran-treated pts continue to experience significant HCRU and costs related to disease progression despite treatment. A need remains for new therapies to further reduce burden and costs."

HEOR • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders

CA.APB.01: Electrophysiology, Amyloidosis & Precision Cardiology (AHA 2025) - "Several studies examine drug safety and efficacy (e.g., tafamidis, acoramidis, vutrisiran), while others investigate mechanisms of cardiac development, pacing strategies, and novel therapeutic approaches like gene transfer. This is not a CE accredited session. Poster Professor: Stavros Stavrakis (UNIVERSITY OF OKLAHOMA)"

Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Infectious Disease • Novel Coronavirus Disease